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Wow..that article was really hard to understand with all the medical

terminology. I am very interested in what that all meant, because that is

the exact type of CMT that we have in our family...and, for me it is

particularly informative because my 5 year old son started showing the

disease at infancy and could be in a wheel chair within the next five

years...if anyone can translate what that all meant...it would be greatly

appreciated. Thanks to you all for everything...Tana

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That article says that in X-linked CMT the first structure to be defective

(bad) is MYELIN, not the axon.

The axon degenerates as a consequence of the myelin defect, not because the

axon was wrong itself.

This was already well-known, because X-linked CMT is due to defective myelin

protein called connexin32: the authors of that article just demonstrated it

again by biopsies on 8 people.

About your 5 year old son, I am sure he will never be in a wheelchair: in my

large CMT population, no CMTX is in a wheelchair. And in the future there

will be a gene therapy to stop the disease.

Paolo Vinci, MD

----- Original Message -----

From: <ann9055@...>

< >

Sent: Saturday, August 04, 2001 6:32 PM

Subject: Re: [] X-linked CMT Article

> Wow..that article was really hard to understand with all the medical

> terminology. I am very interested in what that all meant, because that is

> the exact type of CMT that we have in our family...and, for me it is

> particularly informative because my 5 year old son started showing the

> disease at infancy and could be in a wheel chair within the next five

> years...if anyone can translate what that all meant...it would be greatly

> appreciated. Thanks to you all for everything...Tana

>

>

>

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Dr. Paolo Vinci,

Lamar here,

That may be the symptomology that you have observed but I assure you many

with CMTX are in wheelchairs, including some " symptomatic carriers " While

the CMT in my large family has never been classified as to type and is not

CMTX), I was told at the age of 35 that " You MAY need to use a cane by the

time you are in your 70's " by the MDA neurologist that diagnosed me. I was

in an AFO by my mid 40's, used a cane by my late 40's, and now can only walk

very short distances with a cane or walker. An electric wheelchair has been

ordered. I am 53. Many articles still say CMT is seldom severely

handicapping, and is not life threatening, but many cases show that it can

be both.

In my family well over 50% follow the pattern I have, and most of them

progress to the point that they can not even transfer from a wheelchair to

bed without assistance. Yet some show only mild symptoms for life, and some

require adaptive devices in childhood. Available DNA testing has been

negative, NCV's are only slightly slow but show , " Abnormalities compatible

with a mixed sensory-motor polyneuropathy, having both axonal and

demyelinating characteristics " Biopsy patterns are fairly uniform to:

" There is a severe loss of large diameter myelinated fibers but only a mild

loss of small fibers. Many of the fibers have abnormally thin myelin

sheaths, and a few of them are part of small onion bulbs. Regenerative

clusters of small myelinated fibers are excessive. 56 teased myelinated

fibers were evaluated. 38 of the fibers have increased variation of

internodal lengths consistent with segmental remyelination. 4 of the

myelinated fibers are normal; 3 show uniformly shortened internodes; 1

displays axonal degeneration as reflected as a series of myelin ovoids; and

10 fibers are each closely intertwined with another myelinated fibers,

hindering measurement of internodal lengths.

Diagnosis: Peripheral neuropathy (left sural, biopsy), chronic, moderately

severe with mixed features of axonal degeneration and segmental

remyelination.

Comment: The nerve biopsy specimen demonstrates evidence of segmental

remyelination in 68% of the teased myelinated fibers. This value and the

semithin plastic sections support a chronic demyelinating neuropathy. The

findings are consistent with hereditary motor sensory neuropathies (CMT).

The abnormalities favor the demyelinating type (HMSN, type 1) over HMSN,

type II, despite the reported normal values for motor conduction

velocities. "

It is well known that CMT differs greatly in the extent and rate of

progression with all types, even within families. It has been shown that

identical twins with the same genetic makeup, experience these differences.

With all due respect to your credentials and experience, my 30 years as a

mental health professional make me question if it is psychologically wise to

make the statement you are sure that a child will not require a wheelchair,

when in fact no person has been able to accurately predict the rate or

extent of progression other than it seems to be a pattern of exacerbation

and plateaus. Likewise it is unwise to say that any person will have severe

progression. The glimmer of hope is what keeps many of us going, but to

state they will not need a wheelchair can possibly lead to a big

psychological blow IF the individual does. Many of my own life choices

would have been different had the physician been realistic and not assured

me that my mild symptoms at 35 would not markedly and rapidly progress. I

denied the progression as long as I could, but it rapidly reached the point

I could no longer deny it. I was faced with a sudden change in what I could

do while experiencing a loss of the primary stress reducing (coping)

mechanisms I had always found to work for me. Me being unable to continue

the work I loved due to the potential hazards to my patients, my co-workers,

and myself added to this. Depression followed.

I am not saying that had the doctors not assured me otherwise, these life

changes would have been easier, BUT if they had said, " We don't know, you

may experience only mild symptoms, but you may have marked and crippling

progression, " I would have been better prepared to face what I have to.

As for gene therapy, we all cling to the hope that answers will be found.

But at the same time we must admit that no one knows with certainty when or

if they will be found and approved for use. If the answer is in gene

therapy (which it well may be) I would think that something to either slow

or prevent progression would come well before something that would reverse

existing damage. Remember, they once said Penicillin would make infections

a thing of the past. I cling to the hope that gene therapy or some other

modality will be found to make the life of future generations easier, but at

the age of 53 I must realize that it is highly unlikely that a discovery

will be made and become available in my natural lifetime that will be of

marked benefit for me as an individual. I do feel answers will come. I am

not willing to even guess if they will be 5 years, 10 years, 20 years, 50

years, etc. down the line.

Again, I highly respect you and your work, but feel that the assurances you

gave MAY have been overly optimistic and possibly leading to increased

emotional trauma should this or any individual experience a marked

progression of CMT. The problems that man of us in America have faced for

years has been that most physicians still say that CMT is only mildly

handicapping, does not cause extensive pain, and is never life threatening.

----- Original Message -----

From: Paolo Vinci

Sent: Sunday, August 05, 2001 09:12 AM

Subject: Re: [] X-linked CMT Article

That article says that in X-linked CMT the first structure to be defective

(bad) is MYELIN, not the axon.

The axon degenerates as a consequence of the myelin defect, not because

the

axon was wrong itself.

This was already well-known, because X-linked CMT is due to defective

myelin

protein called connexin32: the authors of that article just demonstrated

it

again by biopsies on 8 people.

About your 5 year old son, I am sure he will never be in a wheelchair: in

my

large CMT population, no CMTX is in a wheelchair. And in the future there

will be a gene therapy to stop the disease.

Paolo Vinci, MD

----- Original Message -----

From: <ann9055@...>

< >

Sent: Saturday, August 04, 2001 6:32 PM

Subject: Re: [] X-linked CMT Article

> Wow..that article was really hard to understand with all the medical

> terminology. I am very interested in what that all meant, because that

is

> the exact type of CMT that we have in our family...and, for me it is

> particularly informative because my 5 year old son started showing the

> disease at infancy and could be in a wheel chair within the next five

> years...if anyone can translate what that all meant...it would be

greatly

> appreciated. Thanks to you all for everything...Tana

>

>

>

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Share on other sites

Guest guest

-----Original Message-----

From: Lamar son <lls@...>

< >

Date: Sunday, August 05, 2001 11:57 AM

Subject: Re: [] X-linked CMT Article

>>>>Thanks Lamar, what you said was what I was going to say to Paolo about

his statement to Tana about her son. Never say never! I cannot see how CMT

can be so different in Italy then it is here can you? I went from very active

to these braces (full leg) in 3 years! Talk about a blow! I was told CMT has

" SLOW " progression and I might need walking aides in my elder years! (as the

Dr. hands me a script for a cane~)

No one can predict how CMT will progress. Even Dr's. Dr's have a tendency to

want to assure a worried patient and that's great, however, tell the patient

what Could happen, then tell the patient that many many CMTer's never have those

things happen, and as hard as it is to say and for the patient to accept, say

we have to just wait and see. And I do hate the wait and see part, but its the

truth and thats better than false hope. Being prepared for anything is a better

way of life, ~>Becky M.

Dr. Paolo Vinci,

Lamar here,

That may be the symptomology that you have observed but I assure you many

with CMTX are in wheelchairs, including some " symptomatic carriers " While

the CMT in my large family has never been classified as to type and is not

CMTX), I was told at the age of 35 that " You MAY need to use a cane by the

time you are in your 70's " by the MDA neurologist that diagnosed me. I was

in an AFO by my mid 40's, used a cane by my late 40's, and now can only walk

very short distances with a cane or walker. An electric wheelchair has been

ordered. I am 53. Many articles still say CMT is seldom severely

handicapping, and is not life threatening, but many cases show that it can

be both.

In my family well over 50% follow the pattern I have, and most of them

progress to the point that they can not even transfer from a wheelchair to

bed without assistance. Yet some show only mild symptoms for life, and some

require adaptive devices in childhood. Available DNA testing has been

negative, NCV's are only slightly slow but show , " Abnormalities compatible

with a mixed sensory-motor polyneuropathy, having both axonal and

demyelinating characteristics " Biopsy patterns are fairly uniform to:

" There is a severe loss of large diameter myelinated fibers but only a mild

loss of small fibers. Many of the fibers have abnormally thin myelin

sheaths, and a few of them are part of small onion bulbs. Regenerative

clusters of small myelinated fibers are excessive. 56 teased myelinated

fibers were evaluated. 38 of the fibers have increased variation of

internodal lengths consistent with segmental remyelination. 4 of the

myelinated fibers are normal; 3 show uniformly shortened internodes; 1

displays axonal degeneration as reflected as a series of myelin ovoids; and

10 fibers are each closely intertwined with another myelinated fibers,

hindering measurement of internodal lengths.

Diagnosis: Peripheral neuropathy (left sural, biopsy), chronic, moderately

severe with mixed features of axonal degeneration and segmental

remyelination.

Comment: The nerve biopsy specimen demonstrates evidence of segmental

remyelination in 68% of the teased myelinated fibers. This value and the

semithin plastic sections support a chronic demyelinating neuropathy. The

findings are consistent with hereditary motor sensory neuropathies (CMT).

The abnormalities favor the demyelinating type (HMSN, type 1) over HMSN,

type II, despite the reported normal values for motor conduction

velocities. "

It is well known that CMT differs greatly in the extent and rate of

progression with all types, even within families. It has been shown that

identical twins with the same genetic makeup, experience these differences.

With all due respect to your credentials and experience, my 30 years as a

mental health professional make me question if it is psychologically wise to

make the statement you are sure that a child will not require a wheelchair,

when in fact no person has been able to accurately predict the rate or

extent of progression other than it seems to be a pattern of exacerbation

and plateaus. Likewise it is unwise to say that any person will have severe

progression. The glimmer of hope is what keeps many of us going, but to

state they will not need a wheelchair can possibly lead to a big

psychological blow IF the individual does. Many of my own life choices

would have been different had the physician been realistic and not assured

me that my mild symptoms at 35 would not markedly and rapidly progress. I

denied the progression as long as I could, but it rapidly reached the point

I could no longer deny it. I was faced with a sudden change in what I could

do while experiencing a loss of the primary stress reducing (coping)

mechanisms I had always found to work for me. Me being unable to continue

the work I loved due to the potential hazards to my patients, my co-workers,

and myself added to this. Depression followed.

I am not saying that had the doctors not assured me otherwise, these life

changes would have been easier, BUT if they had said, " We don't know, you

may experience only mild symptoms, but you may have marked and crippling

progression, " I would have been better prepared to face what I have to.

As for gene therapy, we all cling to the hope that answers will be found.

But at the same time we must admit that no one knows with certainty when or

if they will be found and approved for use. If the answer is in gene

therapy (which it well may be) I would think that something to either slow

or prevent progression would come well before something that would reverse

existing damage. Remember, they once said Penicillin would make infections

a thing of the past. I cling to the hope that gene therapy or some other

modality will be found to make the life of future generations easier, but at

the age of 53 I must realize that it is highly unlikely that a discovery

will be made and become available in my natural lifetime that will be of

marked benefit for me as an individual. I do feel answers will come. I am

not willing to even guess if they will be 5 years, 10 years, 20 years, 50

years, etc. down the line.

Again, I highly respect you and your work, but feel that the assurances you

gave MAY have been overly optimistic and possibly leading to increased

emotional trauma should this or any individual experience a marked

progression of CMT. The problems that man of us in America have faced for

years has been that most physicians still say that CMT is only mildly

handicapping, does not cause extensive pain, and is never life threatening.

----- Original Message -----

From: Paolo Vinci

Sent: Sunday, August 05, 2001 09:12 AM

Subject: Re: [] X-linked CMT Article

That article says that in X-linked CMT the first structure to be defective

(bad) is MYELIN, not the axon.

The axon degenerates as a consequence of the myelin defect, not because

the

axon was wrong itself.

This was already well-known, because X-linked CMT is due to defective

myelin

protein called connexin32: the authors of that article just demonstrated

it

again by biopsies on 8 people.

About your 5 year old son, I am sure he will never be in a wheelchair: in

my

large CMT population, no CMTX is in a wheelchair. And in the future there

will be a gene therapy to stop the disease.

Paolo Vinci, MD

----- Original Message -----

From: <ann9055@...>

< >

Sent: Saturday, August 04, 2001 6:32 PM

Subject: Re: [] X-linked CMT Article

> Wow..that article was really hard to understand with all the medical

> terminology. I am very interested in what that all meant, because that

is

> the exact type of CMT that we have in our family...and, for me it is

> particularly informative because my 5 year old son started showing the

> disease at infancy and could be in a wheel chair within the next five

> years...if anyone can translate what that all meant...it would be

greatly

> appreciated. Thanks to you all for everything...Tana

>

>

>

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Share on other sites

Guest guest

Dear Lamar,

why are you so pessimist?

Don't you think that saying anybody that he will be in a wheelchair in few

years means that he will undergo inevitable depression? Or, in case of

children, the parents will undergo depression!

Better a positive message! Especially if the possibility to need a

wheelchair is minimal.

When I was a boy, my parents were told that I was going to be in a

wheelchair at the age of 18. My mother got depressed and needed medications.

Fortunately I reacted positively and did not care. When I worsened, ten

years

ago, I realized what the causes were (obesity, wrong footwear) and corrected

them: so I could COMPENSATE for the progressive reduction in muscle

strength.

Now, I am still able to walk without a stick.

Doctors can be wrong with diagnosis: I was diagnosed with a variant

of Lou-Gerig disease (a very bad motor neuron disease) by an eminent

professor: I would not be here if I had it.

I see that you have not yet been diagnosed definitevely as to type and

subtype; and that you do not have CMTX, at least not the form due to

connexin32. If your nerve conduction velocities are quite normal, you might

have a CMT2, which can be more severe than most CMT1 cases. In addition many

CMT2 cases present an associated involvement of the central nerve system,

with muscle stiffness and spasms, or other signs which worsen the peripheral

neuropathy (CMT).

There are CMT1 mutations (e.g. P0 124 mutation) and some CMT2

forms (like mine) that can cause severe muscle loss in the lower limbs, with

need of long braces or a wheelchair. There are also recessive forms, which

are severe.

To further complicate the issue, the need of a wheelchair may be due to

personal (obesity, depression, accidental falls, and so on) and

environmental factors I am

studying in order to eliminate them.

I just would like to tell you that, 4 years ago, a nice 14 y.o. girl enter

my

examination room in a wheelchair pushed by her mother. She has CMT 2. She

had been told by my chief that it was better if she did not walk (The less

you walk, the better it is!). It was not easy to visit her, because she was

very depressed and refused any help. She told me that she had NO FUTURE.

I think that, if I too had not had a CMT, she would have never accepted

to undergo rehab.

I visited her: she oscillated and had pain when she stood up for longer than

few seconds. But I found that she had strong quadriceps and glutei muscles!

The reason for her pains was a biomechanical problem in her feet, where an

excessive achilles tendon lengthening had been performed. I corrected her

problem by proper wedges and boots. And she could stand for longer than 10

minutes without oscillations and pain. Now she is 18. I saw her last month.

She walks quite well. She is positive about her future. This year she will

go to the university by public means of transportation.

Was her wheelchair really necessary?

About gene therapy too, why are you so pessimist? In the fifties, many

people died from tubercolosis. Then a drug was found and they lived... they

are still alive and become old.

See you.

Paolo

----- Original Message -----

From: Lamar son <lls@...>

< >

Sent: Sunday, August 05, 2001 7:04 PM

Subject: Re: [] X-linked CMT Article

> Dr. Paolo Vinci,

> Lamar here,

>

> That may be the symptomology that you have observed but I assure you many

> with CMTX are in wheelchairs, including some " symptomatic carriers " While

> the CMT in my large family has never been classified as to type and is not

> CMTX), I was told at the age of 35 that " You MAY need to use a cane by the

> time you are in your 70's " by the MDA neurologist that diagnosed me. I

was

> in an AFO by my mid 40's, used a cane by my late 40's, and now can only

walk

> very short distances with a cane or walker. An electric wheelchair has

been

> ordered. I am 53. Many articles still say CMT is seldom severely

> handicapping, and is not life threatening, but many cases show that it can

> be both.

>

> In my family well over 50% follow the pattern I have, and most of them

> progress to the point that they can not even transfer from a wheelchair to

> bed without assistance. Yet some show only mild symptoms for life, and

some

> require adaptive devices in childhood. Available DNA testing has been

> negative, NCV's are only slightly slow but show , " Abnormalities

compatible

> with a mixed sensory-motor polyneuropathy, having both axonal and

> demyelinating characteristics " Biopsy patterns are fairly uniform to:

> " There is a severe loss of large diameter myelinated fibers but only a

mild

> loss of small fibers. Many of the fibers have abnormally thin myelin

> sheaths, and a few of them are part of small onion bulbs. Regenerative

> clusters of small myelinated fibers are excessive. 56 teased myelinated

> fibers were evaluated. 38 of the fibers have increased variation of

> internodal lengths consistent with segmental remyelination. 4 of the

> myelinated fibers are normal; 3 show uniformly shortened internodes; 1

> displays axonal degeneration as reflected as a series of myelin ovoids;

and

> 10 fibers are each closely intertwined with another myelinated fibers,

> hindering measurement of internodal lengths.

> Diagnosis: Peripheral neuropathy (left sural, biopsy), chronic,

moderately

> severe with mixed features of axonal degeneration and segmental

> remyelination.

> Comment: The nerve biopsy specimen demonstrates evidence of segmental

> remyelination in 68% of the teased myelinated fibers. This value and the

> semithin plastic sections support a chronic demyelinating neuropathy. The

> findings are consistent with hereditary motor sensory neuropathies (CMT).

> The abnormalities favor the demyelinating type (HMSN, type 1) over HMSN,

> type II, despite the reported normal values for motor conduction

> velocities. "

>

> It is well known that CMT differs greatly in the extent and rate of

> progression with all types, even within families. It has been shown that

> identical twins with the same genetic makeup, experience these

differences.

> With all due respect to your credentials and experience, my 30 years as a

> mental health professional make me question if it is psychologically wise

to

> make the statement you are sure that a child will not require a

wheelchair,

> when in fact no person has been able to accurately predict the rate or

> extent of progression other than it seems to be a pattern of exacerbation

> and plateaus. Likewise it is unwise to say that any person will have

severe

> progression. The glimmer of hope is what keeps many of us going, but to

> state they will not need a wheelchair can possibly lead to a big

> psychological blow IF the individual does. Many of my own life choices

> would have been different had the physician been realistic and not assured

> me that my mild symptoms at 35 would not markedly and rapidly progress. I

> denied the progression as long as I could, but it rapidly reached the

point

> I could no longer deny it. I was faced with a sudden change in what I

could

> do while experiencing a loss of the primary stress reducing (coping)

> mechanisms I had always found to work for me. Me being unable to continue

> the work I loved due to the potential hazards to my patients, my

co-workers,

> and myself added to this. Depression followed.

>

> I am not saying that had the doctors not assured me otherwise, these life

> changes would have been easier, BUT if they had said, " We don't know, you

> may experience only mild symptoms, but you may have marked and crippling

> progression, " I would have been better prepared to face what I have to.

>

> As for gene therapy, we all cling to the hope that answers will be found.

> But at the same time we must admit that no one knows with certainty when

or

> if they will be found and approved for use. If the answer is in gene

> therapy (which it well may be) I would think that something to either slow

> or prevent progression would come well before something that would reverse

> existing damage. Remember, they once said Penicillin would make

infections

> a thing of the past. I cling to the hope that gene therapy or some other

> modality will be found to make the life of future generations easier, but

at

> the age of 53 I must realize that it is highly unlikely that a discovery

> will be made and become available in my natural lifetime that will be of

> marked benefit for me as an individual. I do feel answers will come. I

am

> not willing to even guess if they will be 5 years, 10 years, 20 years, 50

> years, etc. down the line.

>

> Again, I highly respect you and your work, but feel that the assurances

you

> gave MAY have been overly optimistic and possibly leading to increased

> emotional trauma should this or any individual experience a marked

> progression of CMT. The problems that man of us in America have faced for

> years has been that most physicians still say that CMT is only mildly

> handicapping, does not cause extensive pain, and is never life

threatening.

> ----- Original Message -----

> From: Paolo Vinci

>

> Sent: Sunday, August 05, 2001 09:12 AM

> Subject: Re: [] X-linked CMT Article

>

>

> That article says that in X-linked CMT the first structure to be

defective

> (bad) is MYELIN, not the axon.

> The axon degenerates as a consequence of the myelin defect, not because

> the

> axon was wrong itself.

> This was already well-known, because X-linked CMT is due to defective

> myelin

> protein called connexin32: the authors of that article just demonstrated

> it

> again by biopsies on 8 people.

> About your 5 year old son, I am sure he will never be in a wheelchair:

in

> my

> large CMT population, no CMTX is in a wheelchair. And in the future

there

> will be a gene therapy to stop the disease.

> Paolo Vinci, MD

>

>

> ----- Original Message -----

> From: <ann9055@...>

> < >

> Sent: Saturday, August 04, 2001 6:32 PM

> Subject: Re: [] X-linked CMT Article

>

>

> > Wow..that article was really hard to understand with all the medical

> > terminology. I am very interested in what that all meant, because

that

> is

> > the exact type of CMT that we have in our family...and, for me it is

> > particularly informative because my 5 year old son started showing the

> > disease at infancy and could be in a wheel chair within the next five

> > years...if anyone can translate what that all meant...it would be

> greatly

> > appreciated. Thanks to you all for everything...Tana

> >

> >

> >

Link to comment
Share on other sites

Guest guest

Sorry Becky, but I cannot agree with Lamar and you. Neither as a patient nor

as a physician.

I probably wasted my youth because of the wheelchair ghost! I am struggling

to avoid that other people live with CMT as if they had a sword on their

neck. Still in absence of a medical treatment, much can be done to improve

functionality and live a normal life. It can and IT MUST. Not only in Italy.

Paolo

----- Original Message -----

From: Maxwell <rmax@...>

< >

Sent: Sunday, August 05, 2001 10:12 PM

Subject: Re: [] X-linked CMT Article

>

> -----Original Message-----

> From: Lamar son <lls@...>

> < >

> Date: Sunday, August 05, 2001 11:57 AM

> Subject: Re: [] X-linked CMT Article

> >>>>Thanks Lamar, what you said was what I was going to say to Paolo

about his statement to Tana about her son. Never say never! I cannot see

how CMT can be so different in Italy then it is here can you? I went from

very active to these braces (full leg) in 3 years! Talk about a blow! I

was told CMT has " SLOW " progression and I might need walking aides in my

elder years! (as the Dr. hands me a script for a cane~)

> No one can predict how CMT will progress. Even Dr's. Dr's have a

tendency to want to assure a worried patient and that's great, however, tell

the patient what Could happen, then tell the patient that many many CMTer's

never have those things happen, and as hard as it is to say and for the

patient to accept, say we have to just wait and see. And I do hate the

wait and see part, but its the truth and thats better than false hope. Being

prepared for anything is a better way of life, ~>Becky M.

>

> Dr. Paolo Vinci,

> Lamar here,

>

> That may be the symptomology that you have observed but I assure you

many

> with CMTX are in wheelchairs, including some " symptomatic carriers "

While

> the CMT in my large family has never been classified as to type and is

not

> CMTX), I was told at the age of 35 that " You MAY need to use a cane by

the

> time you are in your 70's " by the MDA neurologist that diagnosed me. I

was

> in an AFO by my mid 40's, used a cane by my late 40's, and now can only

walk

> very short distances with a cane or walker. An electric wheelchair has

been

> ordered. I am 53. Many articles still say CMT is seldom severely

> handicapping, and is not life threatening, but many cases show that it

can

> be both.

>

> In my family well over 50% follow the pattern I have, and most of them

> progress to the point that they can not even transfer from a wheelchair

to

> bed without assistance. Yet some show only mild symptoms for life, and

some

> require adaptive devices in childhood. Available DNA testing has been

> negative, NCV's are only slightly slow but show , " Abnormalities

compatible

> with a mixed sensory-motor polyneuropathy, having both axonal and

> demyelinating characteristics " Biopsy patterns are fairly uniform to:

> " There is a severe loss of large diameter myelinated fibers but only a

mild

> loss of small fibers. Many of the fibers have abnormally thin myelin

> sheaths, and a few of them are part of small onion bulbs. Regenerative

> clusters of small myelinated fibers are excessive. 56 teased myelinated

> fibers were evaluated. 38 of the fibers have increased variation of

> internodal lengths consistent with segmental remyelination. 4 of the

> myelinated fibers are normal; 3 show uniformly shortened internodes; 1

> displays axonal degeneration as reflected as a series of myelin ovoids;

and

> 10 fibers are each closely intertwined with another myelinated fibers,

> hindering measurement of internodal lengths.

> Diagnosis: Peripheral neuropathy (left sural, biopsy), chronic,

moderately

> severe with mixed features of axonal degeneration and segmental

> remyelination.

> Comment: The nerve biopsy specimen demonstrates evidence of segmental

> remyelination in 68% of the teased myelinated fibers. This value and

the

> semithin plastic sections support a chronic demyelinating neuropathy.

The

> findings are consistent with hereditary motor sensory neuropathies

(CMT).

> The abnormalities favor the demyelinating type (HMSN, type 1) over HMSN,

> type II, despite the reported normal values for motor conduction

> velocities. "

>

> It is well known that CMT differs greatly in the extent and rate of

> progression with all types, even within families. It has been shown

that

> identical twins with the same genetic makeup, experience these

differences.

> With all due respect to your credentials and experience, my 30 years as

a

> mental health professional make me question if it is psychologically

wise to

> make the statement you are sure that a child will not require a

wheelchair,

> when in fact no person has been able to accurately predict the rate or

> extent of progression other than it seems to be a pattern of

exacerbation

> and plateaus. Likewise it is unwise to say that any person will have

severe

> progression. The glimmer of hope is what keeps many of us going, but to

> state they will not need a wheelchair can possibly lead to a big

> psychological blow IF the individual does. Many of my own life choices

> would have been different had the physician been realistic and not

assured

> me that my mild symptoms at 35 would not markedly and rapidly progress.

I

> denied the progression as long as I could, but it rapidly reached the

point

> I could no longer deny it. I was faced with a sudden change in what I

could

> do while experiencing a loss of the primary stress reducing (coping)

> mechanisms I had always found to work for me. Me being unable to

continue

> the work I loved due to the potential hazards to my patients, my

co-workers,

> and myself added to this. Depression followed.

>

> I am not saying that had the doctors not assured me otherwise, these

life

> changes would have been easier, BUT if they had said, " We don't know,

you

> may experience only mild symptoms, but you may have marked and crippling

> progression, " I would have been better prepared to face what I have to.

>

> As for gene therapy, we all cling to the hope that answers will be

found.

> But at the same time we must admit that no one knows with certainty when

or

> if they will be found and approved for use. If the answer is in gene

> therapy (which it well may be) I would think that something to either

slow

> or prevent progression would come well before something that would

reverse

> existing damage. Remember, they once said Penicillin would make

infections

> a thing of the past. I cling to the hope that gene therapy or some

other

> modality will be found to make the life of future generations easier,

but at

> the age of 53 I must realize that it is highly unlikely that a discovery

> will be made and become available in my natural lifetime that will be of

> marked benefit for me as an individual. I do feel answers will come. I

am

> not willing to even guess if they will be 5 years, 10 years, 20 years,

50

> years, etc. down the line.

>

> Again, I highly respect you and your work, but feel that the assurances

you

> gave MAY have been overly optimistic and possibly leading to increased

> emotional trauma should this or any individual experience a marked

> progression of CMT. The problems that man of us in America have faced

for

> years has been that most physicians still say that CMT is only mildly

> handicapping, does not cause extensive pain, and is never life

threatening.

> ----- Original Message -----

> From: Paolo Vinci

>

> Sent: Sunday, August 05, 2001 09:12 AM

> Subject: Re: [] X-linked CMT Article

>

>

> That article says that in X-linked CMT the first structure to be

defective

> (bad) is MYELIN, not the axon.

> The axon degenerates as a consequence of the myelin defect, not

because

> the

> axon was wrong itself.

> This was already well-known, because X-linked CMT is due to defective

> myelin

> protein called connexin32: the authors of that article just

demonstrated

> it

> again by biopsies on 8 people.

> About your 5 year old son, I am sure he will never be in a wheelchair:

in

> my

> large CMT population, no CMTX is in a wheelchair. And in the future

there

> will be a gene therapy to stop the disease.

> Paolo Vinci, MD

>

>

> ----- Original Message -----

> From: <ann9055@...>

> < >

> Sent: Saturday, August 04, 2001 6:32 PM

> Subject: Re: [] X-linked CMT Article

>

>

> > Wow..that article was really hard to understand with all the medical

> > terminology. I am very interested in what that all meant, because

that

> is

> > the exact type of CMT that we have in our family...and, for me it is

> > particularly informative because my 5 year old son started showing

the

> > disease at infancy and could be in a wheel chair within the next

five

> > years...if anyone can translate what that all meant...it would be

> greatly

> > appreciated. Thanks to you all for everything...Tana

> >

> >

> >

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Dr Paolo,

Not pessimistic by any means, but I am realistic. For a physician to grant

false hope about any medical condition is considered to be both ethically

and morally wrong (at least in America). To say that a person with CMTX or

any form of CMT will not experience progression to a point to a point that

they require a wheelchair is very possibly creating false hope. I have yet

to meet a physician that was psychic. You state that you have not seen

patients with CMTX that did require a wheelchair. I feel sure that there

are those on this list that do, and I know that I have met those that do.

As a professional nurse and certified counselor I would be willing to say

that there is a good chance that any child will not require a wheelchair due

to CMT, but that there is an equally realistic chance that they will. You

always encourage activity within safe limits, but it would probably not be

wise for the child to pursue a career as a tight rope walker! (Or any

occupation requiring physical dexterity and leg strength.) I did not heed

my own professional training and was stubborn. I wanted to believe the

physicians that said my CMT would not progress markedly. I relieved stress

by physical activity. I Cleared land by hand and had a small vegetable

garden about 200 ft square. I kept my three acre lawn manicured. I painted

and reroofed my home. I walked on the beach for miles. I climbed 100 ft

pines with spikes and a safety belt to trim them. I hiked in the mountains.

Then my ankles started turning, I started falling, I needed AFO's and then a

cane. Now I can not step up on a curb without a LOT of assistance. I can

not stand even for a few seconds without holding something. I was not

prepared for what I found I was faced with. I would have much preferred the

doctors to have helped prepare me for the POSSIBILITY. I will admit, I

should have known it myself. After all, I have documented CMT in my family

dating back to the 1850's and some 75+% with it have progressed to the point

they could not transfer from a chair to the bed without assistance. Of

course the ancestors in the 1850's were not diagnosed, but at least three

siblings had symptoms that have been present in every generation since and

have descendants with the CMT diagnosis.

Neither am I pessimistic about gene therapy. It could well hold promise in

the future, but if the process was discovered today, it would probably be a

minimum of 10 years before it was accepted for use and many more years

before it would be widely available, and even more before the average

individual with CMT (and possibly a limited income) could afford it. Let's

face it, if a cure for CMT was available today and would cost $250,000 out

of pocket, many of us would have to pass it up. Gene therapy for CMT may

one day be found, but as of today it does not exist. Until it does exist,

and is proven to work and accepted for medical use, and is readily

available; it remains a HOPE and not a certainty.

I will share the story of another young girl. She was only 8 and had

leukemia. Her parents heard of a doctor that had a " cure " . They mortgaged

their home and farm to pay for the cure. She had the treatment and was

(according to the doctor) " cured " . Two weeks later she died. The family

lost not only a child, they lost everything they owned. Today, she may have

survived. Cures for CMT, all cancers, heart disease, HIV etc. are probably

out there in the future, but no one can assure when they will be found. The

stages of grief in a chronic illness or terminal illness are denial, anger,

hope for a miracle, and acceptance. If we stay hung up on hopes for a

miracle we are setting ourselves up for failure. That does not mean to give

up hope, but means that we do not live our life as if something will be

found next week that will make out CMT go away.

I do not know how much or how fast my own CMT will progress, but I am aware

that it MAY become much more severe. I am making an effort to simplify my

life as much as possible. Living alone and having no " caregiver " I know

that it will be up to me to remain independent as long as possible. I

choose not to lead a life of constant dread that I will one day be in a

nursing home, but remain aware that the possibility is realistic and can not

be overlooked.

----- Original Message -----

From: Paolo Vinci

Sent: Monday, August 06, 2001 07:37 AM

Subject: Re: [] X-linked CMT Article

Dear Lamar,

why are you so pessimist?

Don't you think that saying anybody that he will be in a wheelchair in few

years means that he will undergo inevitable depression? Or, in case of

children, the parents will undergo depression!

Better a positive message! Especially if the possibility to need a

wheelchair is minimal.

When I was a boy, my parents were told that I was going to be in a

wheelchair at the age of 18. My mother got depressed and needed

medications.

Fortunately I reacted positively and did not care. When I worsened, ten

years

ago, I realized what the causes were (obesity, wrong footwear) and

corrected

them: so I could COMPENSATE for the progressive reduction in muscle

strength.

Now, I am still able to walk without a stick.

Doctors can be wrong with diagnosis: I was diagnosed with a variant

of Lou-Gerig disease (a very bad motor neuron disease) by an eminent

professor: I would not be here if I had it.

I see that you have not yet been diagnosed definitevely as to type and

subtype; and that you do not have CMTX, at least not the form due to

connexin32. If your nerve conduction velocities are quite normal, you

might

have a CMT2, which can be more severe than most CMT1 cases. In addition

many

CMT2 cases present an associated involvement of the central nerve system,

with muscle stiffness and spasms, or other signs which worsen the

peripheral

neuropathy (CMT).

There are CMT1 mutations (e.g. P0 124 mutation) and some CMT2

forms (like mine) that can cause severe muscle loss in the lower limbs,

with

need of long braces or a wheelchair. There are also recessive forms, which

are severe.

To further complicate the issue, the need of a wheelchair may be due to

personal (obesity, depression, accidental falls, and so on) and

environmental factors I am

studying in order to eliminate them.

I just would like to tell you that, 4 years ago, a nice 14 y.o. girl enter

my

examination room in a wheelchair pushed by her mother. She has CMT 2. She

had been told by my chief that it was better if she did not walk (The less

you walk, the better it is!). It was not easy to visit her, because she

was

very depressed and refused any help. She told me that she had NO FUTURE.

I think that, if I too had not had a CMT, she would have never accepted

to undergo rehab.

I visited her: she oscillated and had pain when she stood up for longer

than

few seconds. But I found that she had strong quadriceps and glutei

muscles!

The reason for her pains was a biomechanical problem in her feet, where an

excessive achilles tendon lengthening had been performed. I corrected her

problem by proper wedges and boots. And she could stand for longer than 10

minutes without oscillations and pain. Now she is 18. I saw her last

month.

She walks quite well. She is positive about her future. This year she will

go to the university by public means of transportation.

Was her wheelchair really necessary?

About gene therapy too, why are you so pessimist? In the fifties, many

people died from tubercolosis. Then a drug was found and they lived...

they

are still alive and become old.

See you.

Paolo

----- Original Message -----

From: Lamar son <lls@...>

< >

Sent: Sunday, August 05, 2001 7:04 PM

Subject: Re: [] X-linked CMT Article

> Dr. Paolo Vinci,

> Lamar here,

>

> That may be the symptomology that you have observed but I assure you

many

> with CMTX are in wheelchairs, including some " symptomatic carriers "

While

> the CMT in my large family has never been classified as to type and is

not

> CMTX), I was told at the age of 35 that " You MAY need to use a cane by

the

> time you are in your 70's " by the MDA neurologist that diagnosed me. I

was

> in an AFO by my mid 40's, used a cane by my late 40's, and now can only

walk

> very short distances with a cane or walker. An electric wheelchair has

been

> ordered. I am 53. Many articles still say CMT is seldom severely

> handicapping, and is not life threatening, but many cases show that it

can

> be both.

>

> In my family well over 50% follow the pattern I have, and most of them

> progress to the point that they can not even transfer from a wheelchair

to

> bed without assistance. Yet some show only mild symptoms for life, and

some

> require adaptive devices in childhood. Available DNA testing has been

> negative, NCV's are only slightly slow but show , " Abnormalities

compatible

> with a mixed sensory-motor polyneuropathy, having both axonal and

> demyelinating characteristics " Biopsy patterns are fairly uniform to:

> " There is a severe loss of large diameter myelinated fibers but only a

mild

> loss of small fibers. Many of the fibers have abnormally thin myelin

> sheaths, and a few of them are part of small onion bulbs. Regenerative

> clusters of small myelinated fibers are excessive. 56 teased myelinated

> fibers were evaluated. 38 of the fibers have increased variation of

> internodal lengths consistent with segmental remyelination. 4 of the

> myelinated fibers are normal; 3 show uniformly shortened internodes; 1

> displays axonal degeneration as reflected as a series of myelin ovoids;

and

> 10 fibers are each closely intertwined with another myelinated fibers,

> hindering measurement of internodal lengths.

> Diagnosis: Peripheral neuropathy (left sural, biopsy), chronic,

moderately

> severe with mixed features of axonal degeneration and segmental

> remyelination.

> Comment: The nerve biopsy specimen demonstrates evidence of segmental

> remyelination in 68% of the teased myelinated fibers. This value and

the

> semithin plastic sections support a chronic demyelinating neuropathy.

The

> findings are consistent with hereditary motor sensory neuropathies

(CMT).

> The abnormalities favor the demyelinating type (HMSN, type 1) over HMSN,

> type II, despite the reported normal values for motor conduction

> velocities. "

>

> It is well known that CMT differs greatly in the extent and rate of

> progression with all types, even within families. It has been shown

that

> identical twins with the same genetic makeup, experience these

differences.

> With all due respect to your credentials and experience, my 30 years as

a

> mental health professional make me question if it is psychologically

wise

to

> make the statement you are sure that a child will not require a

wheelchair,

> when in fact no person has been able to accurately predict the rate or

> extent of progression other than it seems to be a pattern of

exacerbation

> and plateaus. Likewise it is unwise to say that any person will have

severe

> progression. The glimmer of hope is what keeps many of us going, but to

> state they will not need a wheelchair can possibly lead to a big

> psychological blow IF the individual does. Many of my own life choices

> would have been different had the physician been realistic and not

assured

> me that my mild symptoms at 35 would not markedly and rapidly progress.

I

> denied the progression as long as I could, but it rapidly reached the

point

> I could no longer deny it. I was faced with a sudden change in what I

could

> do while experiencing a loss of the primary stress reducing (coping)

> mechanisms I had always found to work for me. Me being unable to

continue

> the work I loved due to the potential hazards to my patients, my

co-workers,

> and myself added to this. Depression followed.

>

> I am not saying that had the doctors not assured me otherwise, these

life

> changes would have been easier, BUT if they had said, " We don't know,

you

> may experience only mild symptoms, but you may have marked and crippling

> progression, " I would have been better prepared to face what I have to.

>

> As for gene therapy, we all cling to the hope that answers will be

found.

> But at the same time we must admit that no one knows with certainty when

or

> if they will be found and approved for use. If the answer is in gene

> therapy (which it well may be) I would think that something to either

slow

> or prevent progression would come well before something that would

reverse

> existing damage. Remember, they once said Penicillin would make

infections

> a thing of the past. I cling to the hope that gene therapy or some

other

> modality will be found to make the life of future generations easier,

but

at

> the age of 53 I must realize that it is highly unlikely that a discovery

> will be made and become available in my natural lifetime that will be of

> marked benefit for me as an individual. I do feel answers will come. I

am

> not willing to even guess if they will be 5 years, 10 years, 20 years,

50

> years, etc. down the line.

>

> Again, I highly respect you and your work, but feel that the assurances

you

> gave MAY have been overly optimistic and possibly leading to increased

> emotional trauma should this or any individual experience a marked

> progression of CMT. The problems that man of us in America have faced

for

> years has been that most physicians still say that CMT is only mildly

> handicapping, does not cause extensive pain, and is never life

threatening.

> ----- Original Message -----

> From: Paolo Vinci

>

> Sent: Sunday, August 05, 2001 09:12 AM

> Subject: Re: [] X-linked CMT Article

>

>

> That article says that in X-linked CMT the first structure to be

defective

> (bad) is MYELIN, not the axon.

> The axon degenerates as a consequence of the myelin defect, not

because

> the

> axon was wrong itself.

> This was already well-known, because X-linked CMT is due to defective

> myelin

> protein called connexin32: the authors of that article just

demonstrated

> it

> again by biopsies on 8 people.

> About your 5 year old son, I am sure he will never be in a wheelchair:

in

> my

> large CMT population, no CMTX is in a wheelchair. And in the future

there

> will be a gene therapy to stop the disease.

> Paolo Vinci, MD

>

>

> ----- Original Message -----

> From: <ann9055@...>

> < >

> Sent: Saturday, August 04, 2001 6:32 PM

> Subject: Re: [] X-linked CMT Article

>

>

> > Wow..that article was really hard to understand with all the medical

> > terminology. I am very interested in what that all meant, because

that

> is

> > the exact type of CMT that we have in our family...and, for me it is

> > particularly informative because my 5 year old son started showing

the

> > disease at infancy and could be in a wheel chair within the next

five

> > years...if anyone can translate what that all meant...it would be

> greatly

> > appreciated. Thanks to you all for everything...Tana

> >

> >

> >

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-----Original Message-----

From: Paolo Vinci <p.vinci@...>

< >

Date: Monday, August 06, 2001 6:46 AM

Subject: Re: [] X-linked CMT Article

Sorry Becky, but I cannot agree with Lamar and you. Neither as a patient nor

as a physician.

>>>The one thing we all can agree upon is, we all have different opinions.

My opinion on it is that, its best to sit the New CMT patient down and tell

them about CMT. Tell them the things that some CMTer's experience, tell them

that some are in wheelchairs, or have to use one for distance. Then tell them,

assure them that even though it can happen, does not mean it will happen to them

or their child. What a shock it is to be told yes you have CMT or your child

has CMT and life is going to be ok, not a problem, just a treatment here and

there some special shoes and everything is going to be ok. That patient may very

well be ok. But that patient may in 5 years be in a wheelchair! And how

shocking when their trusted Dr. didn't tell them a thing about that! That causes

more anger for the CMTer to deal with. And the lost trust of Dr's. And when that

person asks you WHY didn't you let me know this could happen to me? Your answer

of, personally I never thought it would, won't be much help to them. Knowledge

is far from a sword in the neck. It's power, power to over come, to make the

best of, to help avoid, and to prepare ones self just in case it happens, its

not a complete shock which can cause more anger, resentment, fear, frustration,

lack of trust. If it does happen to that patient, how can they ever trust a Dr.

again? And that could emotionaly hang over to other relationships. Job, family,

partner etc. Just my opinion, ~>Becky M.

I probably wasted my youth because of the wheelchair ghost! I am struggling

to avoid that other people live with CMT as if they had a sword on their

neck. Still in absence of a medical treatment, much can be done to improve

functionality and live a normal life. It can and IT MUST. Not only in Italy.

Paolo

----- Original Message -----

From: Maxwell <rmax@...>

< >

Sent: Sunday, August 05, 2001 10:12 PM

Subject: Re: [] X-linked CMT Article

>

> -----Original Message-----

> From: Lamar son <lls@...>

> < >

> Date: Sunday, August 05, 2001 11:57 AM

> Subject: Re: [] X-linked CMT Article

> >>>>Thanks Lamar, what you said was what I was going to say to Paolo

about his statement to Tana about her son. Never say never! I cannot see

how CMT can be so different in Italy then it is here can you? I went from

very active to these braces (full leg) in 3 years! Talk about a blow! I

was told CMT has " SLOW " progression and I might need walking aides in my

elder years! (as the Dr. hands me a script for a cane~)

> No one can predict how CMT will progress. Even Dr's. Dr's have a

tendency to want to assure a worried patient and that's great, however, tell

the patient what Could happen, then tell the patient that many many CMTer's

never have those things happen, and as hard as it is to say and for the

patient to accept, say we have to just wait and see. And I do hate the

wait and see part, but its the truth and thats better than false hope. Being

prepared for anything is a better way of life, ~>Becky M.

>

> Dr. Paolo Vinci,

> Lamar here,

>

> That may be the symptomology that you have observed but I assure you

many

> with CMTX are in wheelchairs, including some " symptomatic carriers "

While

> the CMT in my large family has never been classified as to type and is

not

> CMTX), I was told at the age of 35 that " You MAY need to use a cane by

the

> time you are in your 70's " by the MDA neurologist that diagnosed me. I

was

> in an AFO by my mid 40's, used a cane by my late 40's, and now can only

walk

> very short distances with a cane or walker. An electric wheelchair has

been

> ordered. I am 53. Many articles still say CMT is seldom severely

> handicapping, and is not life threatening, but many cases show that it

can

> be both.

>

> In my family well over 50% follow the pattern I have, and most of them

> progress to the point that they can not even transfer from a wheelchair

to

> bed without assistance. Yet some show only mild symptoms for life, and

some

> require adaptive devices in childhood. Available DNA testing has been

> negative, NCV's are only slightly slow but show , " Abnormalities

compatible

> with a mixed sensory-motor polyneuropathy, having both axonal and

> demyelinating characteristics " Biopsy patterns are fairly uniform to:

> " There is a severe loss of large diameter myelinated fibers but only a

mild

> loss of small fibers. Many of the fibers have abnormally thin myelin

> sheaths, and a few of them are part of small onion bulbs. Regenerative

> clusters of small myelinated fibers are excessive. 56 teased myelinated

> fibers were evaluated. 38 of the fibers have increased variation of

> internodal lengths consistent with segmental remyelination. 4 of the

> myelinated fibers are normal; 3 show uniformly shortened internodes; 1

> displays axonal degeneration as reflected as a series of myelin ovoids;

and

> 10 fibers are each closely intertwined with another myelinated fibers,

> hindering measurement of internodal lengths.

> Diagnosis: Peripheral neuropathy (left sural, biopsy), chronic,

moderately

> severe with mixed features of axonal degeneration and segmental

> remyelination.

> Comment: The nerve biopsy specimen demonstrates evidence of segmental

> remyelination in 68% of the teased myelinated fibers. This value and

the

> semithin plastic sections support a chronic demyelinating neuropathy.

The

> findings are consistent with hereditary motor sensory neuropathies

(CMT).

> The abnormalities favor the demyelinating type (HMSN, type 1) over HMSN,

> type II, despite the reported normal values for motor conduction

> velocities. "

>

> It is well known that CMT differs greatly in the extent and rate of

> progression with all types, even within families. It has been shown

that

> identical twins with the same genetic makeup, experience these

differences.

> With all due respect to your credentials and experience, my 30 years as

a

> mental health professional make me question if it is psychologically

wise to

> make the statement you are sure that a child will not require a

wheelchair,

> when in fact no person has been able to accurately predict the rate or

> extent of progression other than it seems to be a pattern of

exacerbation

> and plateaus. Likewise it is unwise to say that any person will have

severe

> progression. The glimmer of hope is what keeps many of us going, but to

> state they will not need a wheelchair can possibly lead to a big

> psychological blow IF the individual does. Many of my own life choices

> would have been different had the physician been realistic and not

assured

> me that my mild symptoms at 35 would not markedly and rapidly progress.

I

> denied the progression as long as I could, but it rapidly reached the

point

> I could no longer deny it. I was faced with a sudden change in what I

could

> do while experiencing a loss of the primary stress reducing (coping)

> mechanisms I had always found to work for me. Me being unable to

continue

> the work I loved due to the potential hazards to my patients, my

co-workers,

> and myself added to this. Depression followed.

>

> I am not saying that had the doctors not assured me otherwise, these

life

> changes would have been easier, BUT if they had said, " We don't know,

you

> may experience only mild symptoms, but you may have marked and crippling

> progression, " I would have been better prepared to face what I have to.

>

> As for gene therapy, we all cling to the hope that answers will be

found.

> But at the same time we must admit that no one knows with certainty when

or

> if they will be found and approved for use. If the answer is in gene

> therapy (which it well may be) I would think that something to either

slow

> or prevent progression would come well before something that would

reverse

> existing damage. Remember, they once said Penicillin would make

infections

> a thing of the past. I cling to the hope that gene therapy or some

other

> modality will be found to make the life of future generations easier,

but at

> the age of 53 I must realize that it is highly unlikely that a discovery

> will be made and become available in my natural lifetime that will be of

> marked benefit for me as an individual. I do feel answers will come. I

am

> not willing to even guess if they will be 5 years, 10 years, 20 years,

50

> years, etc. down the line.

>

> Again, I highly respect you and your work, but feel that the assurances

you

> gave MAY have been overly optimistic and possibly leading to increased

> emotional trauma should this or any individual experience a marked

> progression of CMT. The problems that man of us in America have faced

for

> years has been that most physicians still say that CMT is only mildly

> handicapping, does not cause extensive pain, and is never life

threatening.

> ----- Original Message -----

> From: Paolo Vinci

>

> Sent: Sunday, August 05, 2001 09:12 AM

> Subject: Re: [] X-linked CMT Article

>

>

> That article says that in X-linked CMT the first structure to be

defective

> (bad) is MYELIN, not the axon.

> The axon degenerates as a consequence of the myelin defect, not

because

> the

> axon was wrong itself.

> This was already well-known, because X-linked CMT is due to defective

> myelin

> protein called connexin32: the authors of that article just

demonstrated

> it

> again by biopsies on 8 people.

> About your 5 year old son, I am sure he will never be in a wheelchair:

in

> my

> large CMT population, no CMTX is in a wheelchair. And in the future

there

> will be a gene therapy to stop the disease.

> Paolo Vinci, MD

>

>

> ----- Original Message -----

> From: <ann9055@...>

> < >

> Sent: Saturday, August 04, 2001 6:32 PM

> Subject: Re: [] X-linked CMT Article

>

>

> > Wow..that article was really hard to understand with all the medical

> > terminology. I am very interested in what that all meant, because

that

> is

> > the exact type of CMT that we have in our family...and, for me it is

> > particularly informative because my 5 year old son started showing

the

> > disease at infancy and could be in a wheel chair within the next

five

> > years...if anyone can translate what that all meant...it would be

> greatly

> > appreciated. Thanks to you all for everything...Tana

> >

> >

> >

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Lamar,

I certainly do not look on your words as pessimistic. Anyone who has

dealt with CMT for years knows that we 'don't know' what is in our

future. But we do not look on the dark side--there is always hope.

My first symptoms started when I was 10-11 years old--mainly falling, and

spraining an ankle. At that time, I put down my 'doppiness' as just

taking after my father, who had much trouble walking and keeping his

balance in later years.

My CMT1A was not dx until I was 39 (I'm now 73),but by that time, I was

married and had four children, three of whom have inherited it in varying

degrees.

I now use a cane, and a w/c when I know there will be some walking

required. Like you, I can not get up a curb by myself, even with a cane,

but have learned to wait until someone comes by and ask if they'll help

me--I have never been turned down. The funny thing is, sometimes it's

someone older than me who offers to help.

I did seem to have a 'dormant' period during my 40's and into the 50's,

when it seemed to get no worse, but now it seems to be progressing very

fast. But I am at a stage in my life when I can take things slow and

easy, thank goodness. I feel for those of us who are younger and still

raising a family, if their symptoms are severe--they are in my daily

prayers.

We've learned so much about CMT in the last decade or so, and still have

so much more to learn.

To a CMT-free future--Theresa

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-----Original Message-----

From: Benj. S Ember <benember@...>

< >

Date: Monday, August 06, 2001 4:51 PM

Subject: Re: [] X-linked CMT Article

Lamar,

I certainly do not look on your words as pessimistic. Anyone who has

dealt with CMT for years knows that we 'don't know' what is in our

future. But we do not look on the dark side--there is always hope.

To a CMT-free future--Theresa

>>>>Theresa, DITTO! Lamar was far from being pessimistic in his post. Just

stating facts. None of us know our futures with or with out CMT. And to state

otherwise when dealing with a disability? Well, it just can't be done! Or

shouldn't be done. Awareness of what might happen is better than having hopes

smashed. At least one knows IF it

ever happens, that it could of happened to them. ~>Becky M.

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