Going to give CARNI-VAL a whirl

[Guest guest]

A neurgolist here in the Twin Cities said he would be willing to

prescribe CARNI-VALfor me. They've asked for a blood-test to see how

many back-up copies of SMN2 I have. If enough (I don't know what the

number is) they'll put me on the drug.

Any pointers from those that have tried this approach?

[Guest guest]

I never heared about it. Can you give me a link / information about it?

Thanks,

best wishes,

Camilla

>

> A neurgolist here in the Twin Cities said he would be willing to

> prescribe CARNI-VALfor me. They've asked for a blood-test to see how

> many back-up copies of SMN2 I have. If enough (I don't know what the

> number is) they'll put me on the drug.

>

> Any pointers from those that have tried this approach?

>

>

>

[Guest guest]

Bri-guy:

I don't see what your SMN2 gene copy-count has to do

with whether or not a doctor prescribes you VPA.

Based on all sorts of studies on VPA & SMA - VPA acts

on any number of SMN2 genes and - they think - causes

more exon 7 & 8 inclusion in the protein production

(SMN2 produces primarilly the shortened SMN Delta 7

protein). The main concerns with VPA are/should be

monitoring possible side effects first (because in the

most extreme - can kill you) - regular blood tests -

every 4 to 6 months should accompany anyone taking VPA

- off-lable or not.

Supposed " Able Bodied " people have 2 copies of both

SMN1 and SMN2 genes. Type 1 SMA is usually a result

of 1 or 2 copies of the SMN2 gene. TYpe 2 SMA

typically has from 2 to 4 copies of SMN2. Type 3 SMA

usually have 3 or more copies of SMN2. I don't

believe the current CarniVal trial has an SMN2 copy

count criteria - thus, I see no reason for your

neurologist to impose one on you.

If you do go forward with it - I hope it helps you!

Mitch-Aurora, OH

(I've got a very close college friend named who

now lives in Minneapolis - and I refer to him as

Bry-guy - sorry, I couldn't resist with Bri-guy - I am

a creature of habit)

--- <boojiboy@...> wrote:

> A neurgolist here in the Twin Cities said he would

> be willing to

> prescribe CARNI-VALfor me. They've asked for a

> blood-test to see how

> many back-up copies of SMN2 I have. If enough (I

> don't know what the

> number is) they'll put me on the drug.

>

> Any pointers from those that have tried this

> approach?

>

>

>

>

>

>

__________________________________________________

[Guest guest]

Here's a good link:

http://www.fsma.org/clin_trials_quest06.shtml

The trials are only for children. However, some doctors are willing

to let adults try it.

> >

> > A neurgolist here in the Twin Cities said he would be willing to

> > prescribe CARNI-VALfor me. They've asked for a blood-test to

see how

> > many back-up copies of SMN2 I have. If enough (I don't know

what the

> > number is) they'll put me on the drug.

> >

> > Any pointers from those that have tried this approach?

> >

> >

> >

>

[Guest guest]

Great info Mitch. I'll bring your post to my doctors appointment

for good discussion point.

I believe they are under the assumption that the results are better

with a higher SMN2 count. But what you say makes sense too.

Thanks for the well wishes, I'll definitely do the routine blood-

tests to make sure nothing nasty is happening. I don't hold out

high hopes, but it worth a shot.

>

> > A neurgolist here in the Twin Cities said he would

> > be willing to

> > prescribe CARNI-VALfor me. They've asked for a

> > blood-test to see how

> > many back-up copies of SMN2 I have. If enough (I

> > don't know what the

> > number is) they'll put me on the drug.

> >

> > Any pointers from those that have tried this

> > approach?

> >

> >

> >

> >

> >

> >

>

>

> __________________________________________________

>

[Guest guest]

Bri-guy:

It is correct to assume the more copies of the SMN2

gene, the larger the possible response of VPA usage

would bring. Larger does not mean " more important. "

Any increase - be it large or small - in flSMN protein

MUST be a VERY GOOD thing for anyone with a

disease/condition caused by a lack of it.

If taking VPA or some other HDAC drug or even some

other method of increasing the protein production -

via RNA interference, gene therapy, etc. - doesn't

matter how - or even how much necessarily in my

oppinion - IF IT HELPS SOMEONE in some way - be it big

or small (and who's to judge the level of importance)

- than it is certainly worthwhile to at least be

allowed to make the decision of whether to give it a

shot or not.

Yes the results are " better " if better only means

more. " Better " for you is completely independent than

" better " is to anyone else. If you have the courage

to individually trial an FDA approved medication

off-label, it should not be hindered by other's

definition of the word " better. "

If I was at that doctors office - I'd tell them to

stick their SMN2 copy count criteria where the sun

don't shine. Or " up their nose with a rubber hose " (a

good Fonzie quote for you)...

Good luck - Mitch

--- <boojiboy@...> wrote:

> Great info Mitch. I'll bring your post to my doctors

> appointment

> for good discussion point.

>

> I believe they are under the assumption that the

> results are better

> with a higher SMN2 count. But what you say makes

> sense too.

>

> Thanks for the well wishes, I'll definitely do the

> routine blood-

> tests to make sure nothing nasty is happening. I

> don't hold out

> high hopes, but it worth a shot.

>

>

>

>

> >

> > > A neurgolist here in the Twin Cities said he

> would

> > > be willing to

> > > prescribe CARNI-VALfor me. They've asked for a

> > > blood-test to see how

> > > many back-up copies of SMN2 I have. If enough

> (I

> > > don't know what the

> > > number is) they'll put me on the drug.

> > >

> > > Any pointers from those that have tried this

> > > approach?

> > >

> > >

> > >

> > >

> > >

> > >

> >

> >

> > __________________________________________________

> >

[Guest guest]

Mitch, I like your attitude.

I sent a note to the geneticist at the clinic asking about the

concern over SMN2 copy. She responded already stating that a small

portion of individuals have no SMN2 copies at all. In these rare

cases VPA would have no effect.

So bottom line, if they are going to put me at risk of potential

side-effects, then they want to be certain I have at least a chance

of benefit from VPA. Makes sense.

I never knew a SMN2 count could be 0. Especially for a Type II like

me. Interesting.

> > >

> > > > A neurgolist here in the Twin Cities said he

> > would

> > > > be willing to

> > > > prescribe CARNI-VALfor me. They've asked for a

> > > > blood-test to see how

> > > > many back-up copies of SMN2 I have. If enough

> > (I

> > > > don't know what the

> > > > number is) they'll put me on the drug.

> > > >

> > > > Any pointers from those that have tried this

> > > > approach?

> > > >

> > > >

> > > >

> > > >

> > > >

> > > >

> > >

> > >

> > > __________________________________________________

> > >

[Guest guest]

I think they're wrong on that. The only people I know

of with zero copies of both SMN1 & SMN2 genes are

still-born or miscarried - at least based on what I've

read or discussed with doctors, etc. Don't get me

wrong, I'm not geneticist - I'm just a dumb guy dad

with a business degree who's read a couple books, but

that response you were sent below by the geneticist to

me sounds pretty lame-brain to me.

Thus, I see no reason for them to deny you your choice

in this matter - because you certainly have more than

0 copies of SMN2 gene.

--- <boojiboy@...> wrote:

> Mitch, I like your attitude.

>

> I sent a note to the geneticist at the clinic asking

> about the

> concern over SMN2 copy. She responded already

> stating that a small

> portion of individuals have no SMN2 copies at all.

> In these rare

> cases VPA would have no effect.

>

> So bottom line, if they are going to put me at risk

> of potential

> side-effects, then they want to be certain I have at

> least a chance

> of benefit from VPA. Makes sense.

>

> I never knew a SMN2 count could be 0. Especially

> for a Type II like

> me. Interesting.

>

>

>

>

> > > >

> > > > > A neurgolist here in the Twin Cities said he

> > > would

> > > > > be willing to

> > > > > prescribe CARNI-VALfor me. They've asked

> for a

> > > > > blood-test to see how

> > > > > many back-up copies of SMN2 I have. If

> enough

> > > (I

> > > > > don't know what the

> > > > > number is) they'll put me on the drug.

> > > > >

> > > > > Any pointers from those that have tried this

> > > > > approach?

> > > > >

> > > > >

> > > > >

> > > > >

> > > > >

> > > > >

> > > >

> > > >

> > > >

> __________________________________________________

> > > >

[Guest guest]

From what I understand the blood test is for a liver function test to see if

your liver can handle the medication. I am using the drug now and not with the

carnitine because the liver damage is rare and usually only happens to very

young children 2 and under.

I have to have my blood tested every few months to check my liver just to

make sure no damage is being done.

<boojiboy@...> wrote:

Great info Mitch. I'll bring your post to my doctors appointment

for good discussion point.

I believe they are under the assumption that the results are better

with a higher SMN2 count. But what you say makes sense too.

Thanks for the well wishes, I'll definitely do the routine blood-

tests to make sure nothing nasty is happening. I don't hold out

high hopes, but it worth a shot.

>

> > A neurgolist here in the Twin Cities said he would

> > be willing to

> > prescribe CARNI-VALfor me. They've asked for a

> > blood-test to see how

> > many back-up copies of SMN2 I have. If enough (I

> > don't know what the

> > number is) they'll put me on the drug.

> >

> > Any pointers from those that have tried this

> > approach?

> >

> >

> >

> >

> >

> >

>

>

> __________________________________________________

>

[Guest guest]

Hi Joy:

I hope your personal trial of the medication helps

you! Regarding the carnitine - and why its part of

the " Carni-Val " trial therapy - it isn't because of

liver considerations, though you are correct in that

liver complications with VPA are some of the biggest

concerns. Carnitine -as naturally produced by one's

own body, it seems, was being depleted due to VPA

usage. Natural carnitine levels, I believe, in some

of the children early on in the Phase 1 trial were

apparently being depleted due to VPA usage - and in

blood tests showed extreme low levels of carnitine.

Low carnitine levels can cause increasing problems

with the functioning of one's muscles (so I'm told) -

thus potentially off-setting any gains due to VPA

usage. I would make sure your neurologist carefully

monitor your carnitine levels along with liver

functions ( & all else they look at) via regular blood

draws. Additionally - I think you should seriously

consider adding a carnitine supplement to your diet or

medication. Good luck - Mitch

--- Joy <j0yeuxx@...> wrote:

> From what I understand the blood test is for a liver

> function test to see if your liver can handle the

> medication. I am using the drug now and not with the

> carnitine because the liver damage is rare and

> usually only happens to very young children 2 and

> under.

> I have to have my blood tested every few months

> to check my liver just to make sure no damage is

> being done.

>

> <boojiboy@...> wrote:

> Great info Mitch. I'll bring your post to my

> doctors appointment

> for good discussion point.

>

> I believe they are under the assumption that the

> results are better

> with a higher SMN2 count. But what you say makes

> sense too.

>

> Thanks for the well wishes, I'll definitely do the

> routine blood-

> tests to make sure nothing nasty is happening. I

> don't hold out

> high hopes, but it worth a shot.

>

>

>

>

> >

> > > A neurgolist here in the Twin Cities said he

> would

> > > be willing to

> > > prescribe CARNI-VALfor me. They've asked for a

> > > blood-test to see how

> > > many back-up copies of SMN2 I have. If enough (I

> > > don't know what the

> > > number is) they'll put me on the drug.

> > >

> > > Any pointers from those that have tried this

> > > approach?

> > >

> > >

> > >

> > >

> > >

> > >

> >

> >

> > __________________________________________________

> >

[Guest guest]

I'm also just a dumb guy who happens to read every piece of info on

SMA that I can find, and everything I have read says that Mitch is

right. Animal studies of SMA have shown that humans seem to be the

only animal with any SMN2 genes at all. Animals only have SMN1, and if

you take that away they die in the womb. Someone who has SMA (which by

definition means no SMN1 gene) and also has no SMN2 copies would not

be able to survive. If you have SMA Type 2, you must have some SMN

protein, therefore you must have at least one copy of SMN2, therefore

VPA might help.

This article about VPA in Type 3 adults mentions the SMN2 issue. It

might be true that the more SMN2 copies you have, the more VPA will

increase the protein, but even a small increase may be beneficial for

someone with very little to start with.

http://tinyurl.com/zol7t

Good luck!

2006/8/11, <mongomustgolf@...>:

> I think they're wrong on that. The only people I know

> of with zero copies of both SMN1 & SMN2 genes are

> still-born or miscarried - at least based on what I've

> read or discussed with doctors, etc. Don't get me

> wrong, I'm not geneticist - I'm just a dumb guy dad

> with a business degree who's read a couple books, but

> that response you were sent below by the geneticist to

> me sounds pretty lame-brain to me.

>

> Thus, I see no reason for them to deny you your choice

> in this matter - because you certainly have more than

> 0 copies of SMN2 gene.

>

[Guest guest]

Great article. Thanks.

Yeah, it does makes sense that if I had no SMN2 I'd be dead. I'll

let them get the results of the blood test and wait until my

appointment to ask the doctor about it.

>

> I'm also just a dumb guy who happens to read every piece of info on

> SMA that I can find, and everything I have read says that Mitch is

> right. Animal studies of SMA have shown that humans seem to be the

> only animal with any SMN2 genes at all. Animals only have SMN1,

and if

> you take that away they die in the womb. Someone who has SMA

(which by

> definition means no SMN1 gene) and also has no SMN2 copies would

not

> be able to survive. If you have SMA Type 2, you must have some SMN

> protein, therefore you must have at least one copy of SMN2,

therefore

> VPA might help.

>

> This article about VPA in Type 3 adults mentions the SMN2 issue. It

> might be true that the more SMN2 copies you have, the more VPA will

> increase the protein, but even a small increase may be beneficial

for

> someone with very little to start with.

> http://tinyurl.com/zol7t

>

> Good luck!

>

>

>

>

> 2006/8/11, <mongomustgolf@...>:

> > I think they're wrong on that. The only people I know

> > of with zero copies of both SMN1 & SMN2 genes are

> > still-born or miscarried - at least based on what I've

> > read or discussed with doctors, etc. Don't get me

> > wrong, I'm not geneticist - I'm just a dumb guy dad

> > with a business degree who's read a couple books, but

> > that response you were sent below by the geneticist to

> > me sounds pretty lame-brain to me.

> >

> > Thus, I see no reason for them to deny you your choice

> > in this matter - because you certainly have more than

> > 0 copies of SMN2 gene.

> >

>

[Guest guest]

If an animal does not have SMN2 genes, how can we use

SMA mouses or Zebra animal models to test a drug which

might potentially up-regulate SMN2 genes to make more

SMN protein?

--- Senior <jsenior@...> wrote:

> I'm also just a dumb guy who happens to read every

> piece of info on

> SMA that I can find, and everything I have read says

> that Mitch is

> right. Animal studies of SMA have shown that humans

> seem to be the

> only animal with any SMN2 genes at all. Animals only

> have SMN1, and if

> you take that away they die in the womb. Someone who

> has SMA (which by

> definition means no SMN1 gene) and also has no SMN2

> copies would not

> be able to survive. If you have SMA Type 2, you must

> have some SMN

> protein, therefore you must have at least one copy

> of SMN2, therefore

> VPA might help.

>

> This article about VPA in Type 3 adults mentions the

> SMN2 issue. It

> might be true that the more SMN2 copies you have,

> the more VPA will

> increase the protein, but even a small increase may

> be beneficial for

> someone with very little to start with.

> http://tinyurl.com/zol7t

>

> Good luck!

>

>

>

>

> 2006/8/11,

> <mongomustgolf@...>:

> > I think they're wrong on that. The only people I

> know

> > of with zero copies of both SMN1 & SMN2 genes are

> > still-born or miscarried - at least based on what

> I've

> > read or discussed with doctors, etc. Don't get me

> > wrong, I'm not geneticist - I'm just a dumb guy

> dad

> > with a business degree who's read a couple books,

> but

> > that response you were sent below by the

> geneticist to

> > me sounds pretty lame-brain to me.

> >

> > Thus, I see no reason for them to deny you your

> choice

> > in this matter - because you certainly have more

> than

> > 0 copies of SMN2 gene.

> >

>

__________________________________________________

[Guest guest]

I got my SMN2 gene copy test back. They called in inconclusive! My

geneticist said that this probably means they weren't willing to

make the call between 0 or 1 copy, 2 and 3 copies, etc. etc.

I find this odd since they wanted to ensure I had at least one copy

ensuring that VPA would at least have a chance at benefit.

I had the discussion again stating that if I had 0 copies, I

wouldn't be here asking about it. Once again, the geneticist

emphasized that they do indeed get patients with 0 copies. She said

that in these cases the SMN gene isn't actually deleted, but instead

is defective, partially deleted or what she called, 'migrated'.

They are going to go ahead with VPA with me and redo the test later.

Thought folks would be interested in this little tidbit of info.

> >

> > > I'm also just a dumb guy who happens to read every

> > > piece of info on

> > > SMA that I can find, and everything I have read says

> > > that Mitch is

> > > right. Animal studies of SMA have shown that humans

> > > seem to be the

> > > only animal with any SMN2 genes at all. Animals only

> > > have SMN1, and if

> > > you take that away they die in the womb. Someone who

> > > has SMA (which by

> > > definition means no SMN1 gene) and also has no SMN2

> > > copies would not

> > > be able to survive. If you have SMA Type 2, you must

> > > have some SMN

> > > protein, therefore you must have at least one copy

> > > of SMN2, therefore

> > > VPA might help.

> > >

> > > This article about VPA in Type 3 adults mentions the

> > > SMN2 issue. It

> > > might be true that the more SMN2 copies you have,

> > > the more VPA will

> > > increase the protein, but even a small increase may

> > > be beneficial for

> > > someone with very little to start with.

> > > http://tinyurl.com/zol7t

> > >

> > > Good luck!

> > >

>

[Guest guest]

Bri-guy:

Interesting. That geneticist should talk with someone

part of Project Cure (CariVal trial) or a member of

AmSMArt or some knoweldgeable MDA doc - if you want to

give them my name and email - I'll gladly direct them

to some helpful doctor with more knowledge than I

posses. SMN1 and SMN2 genes are identical with one

very small exception in exon 7 of the SMN2 gene which

causes the protein that is coded to not include

various elements coded under exon 7 & 8 (SMN delta 7

protein). From what I've read, most seem to think the

SMN2 gene originated as a mutated form of the SMN1

gene.

I suppose if the SMN1 gene is mutated some other way

than to cause it appear as an SMN2 gene - and it is

still capable of producing some amount of flSMNprotein

(which seems unlikely if the gene has been partly

deleted or 'migrated') - along with having no SMN2

genes (note the SMN2 gene is still able to produce

flSMN protein ~10% of the time despite the coding

error), that someone could indeed have SMA - though

I've not heard of this.

Regardless, best wishes for you - I hope the trial

proves beneficial for you!

Mitch

--- <boojiboy@...> wrote:

> I got my SMN2 gene copy test back. They called in

> inconclusive! My

> geneticist said that this probably means they

> weren't willing to

> make the call between 0 or 1 copy, 2 and 3 copies,

> etc. etc.

>

> I find this odd since they wanted to ensure I had at

> least one copy

> ensuring that VPA would at least have a chance at

> benefit.

>

> I had the discussion again stating that if I had 0

> copies, I

> wouldn't be here asking about it. Once again, the

> geneticist

> emphasized that they do indeed get patients with 0

> copies. She said

> that in these cases the SMN gene isn't actually

> deleted, but instead

> is defective, partially deleted or what she called,

> 'migrated'.

>

> They are going to go ahead with VPA with me and redo

> the test later.

>

> Thought folks would be interested in this little

> tidbit of info.

>

>

>

>

>

> > >

> > > > I'm also just a dumb guy who happens to read

> every

> > > > piece of info on

> > > > SMA that I can find, and everything I have

> read says

> > > > that Mitch is

> > > > right. Animal studies of SMA have shown that

> humans

> > > > seem to be the

> > > > only animal with any SMN2 genes at all.

> Animals only

> > > > have SMN1, and if

> > > > you take that away they die in the womb.

> Someone who

> > > > has SMA (which by

> > > > definition means no SMN1 gene) and also has

> no SMN2

> > > > copies would not

> > > > be able to survive. If you have SMA Type 2,

> you must

> > > > have some SMN

> > > > protein, therefore you must have at least one

> copy

> > > > of SMN2, therefore

> > > > VPA might help.

> > > >

> > > > This article about VPA in Type 3 adults

> mentions the

> > > > SMN2 issue. It

> > > > might be true that the more SMN2 copies you

> have,

> > > > the more VPA will

> > > > increase the protein, but even a small

> increase may

> > > > be beneficial for

> > > > someone with very little to start with.

> > > > http://tinyurl.com/zol7t

> > > >

> > > > Good luck!

> > > >

> >

>

>

>

>

>

>

__________________________________________________

[Guest guest]

Time to resurrect this thread with an update. I met with my

neurologist today and he was very open to the VPA treatment.

However, he ultimately talked me into delaying it for 4-6 months.

It's his belief that there may be information soon to lead

researchers to prescribe sodium phenylbutyrate instead of VPA. In

addition, he believes there will be a coordinated trial for adults

underway soon that I could participate in. This way, I get the

medicine AND I'm being tracked according to the protocol of the

trial.

Finally, and I admit what I am about to say is technically over my

head because I haven't looked into it yet. But, my neurologist

talked about histone deacetylase (HDAC)inhibitors. This is what VPA

and sodium phenylbutyrate are. He believe there was a new

generation of these specific inhibitors in the works and that the

understanding of those inhibitors and their impact on diseases like

SMA is grown a lot in the past couple years.

So, none of this is probably new for those of you that keep abreast

of research. But, I'll set up another appointment for 6 months from

now and see what's cooking. In the meantime, he prescribed

Mestodone (sp?) for a little energy boost when I need it. He

thought it worked for about half the folks who try it.

> > > >

> > > > > I'm also just a dumb guy who happens to read

> > every

> > > > > piece of info on

> > > > > SMA that I can find, and everything I have

> > read says

> > > > > that Mitch is

> > > > > right. Animal studies of SMA have shown that

> > humans

> > > > > seem to be the

> > > > > only animal with any SMN2 genes at all.

> > Animals only

> > > > > have SMN1, and if

> > > > > you take that away they die in the womb.

> > Someone who

> > > > > has SMA (which by

> > > > > definition means no SMN1 gene) and also has

> > no SMN2

> > > > > copies would not

> > > > > be able to survive. If you have SMA Type 2,

> > you must

> > > > > have some SMN

> > > > > protein, therefore you must have at least one

> > copy

> > > > > of SMN2, therefore

> > > > > VPA might help.

> > > > >

> > > > > This article about VPA in Type 3 adults

> > mentions the

> > > > > SMN2 issue. It

> > > > > might be true that the more SMN2 copies you

> > have,

> > > > > the more VPA will

> > > > > increase the protein, but even a small

> > increase may

> > > > > be beneficial for

> > > > > someone with very little to start with.

> > > > > http://tinyurl.com/zol7t

> > > > >

> > > > > Good luck!

> > > > >

> > >

> >

> >

> >

> >

> >

> >

>

>

> __________________________________________________

>

[Guest guest]

,

Could it be Mestinon?

If yes, then that is what I take every day!!!!!!!!

I fiddled around with the dosage until I found one that gave me the most boost

with the least amount of side effect; for me it's 30mg or half a pill

mornings. I take it while doing my " business on the bed pan " so I am relaxed

while my med soaks into my system.

I can do without it, but it lessens my frustrations when moving about.

Angie

On Monday 23 October 2006 19:41, wrote:

> Time to resurrect this thread with an update. I met with my

> neurologist today and he was very open to the VPA treatment.

> However, he ultimately talked me into delaying it for 4-6 months.

>

> It's his belief that there may be information soon to lead

> researchers to prescribe sodium phenylbutyrate instead of VPA. In

> addition, he believes there will be a coordinated trial for adults

> underway soon that I could participate in. This way, I get the

> medicine AND I'm being tracked according to the protocol of the

> trial.

>

> Finally, and I admit what I am about to say is technically over my

> head because I haven't looked into it yet. But, my neurologist

> talked about histone deacetylase (HDAC)inhibitors. This is what VPA

> and sodium phenylbutyrate are. He believe there was a new

> generation of these specific inhibitors in the works and that the

> understanding of those inhibitors and their impact on diseases like

> SMA is grown a lot in the past couple years.

>

> So, none of this is probably new for those of you that keep abreast

> of research. But, I'll set up another appointment for 6 months from

> now and see what's cooking. In the meantime, he prescribed

> Mestodone (sp?) for a little energy boost when I need it. He

> thought it worked for about half the folks who try it.

>

>

>

>

> > > > > > I'm also just a dumb guy who happens to read

> > >

> > > every

> > >

> > > > > > piece of info on

> > > > > > SMA that I can find, and everything I have

> > >

> > > read says

> > >

> > > > > > that Mitch is

> > > > > > right. Animal studies of SMA have shown that

> > >

> > > humans

> > >

> > > > > > seem to be the

> > > > > > only animal with any SMN2 genes at all.

> > >

> > > Animals only

> > >

> > > > > > have SMN1, and if

> > > > > > you take that away they die in the womb.

> > >

> > > Someone who

> > >

> > > > > > has SMA (which by

> > > > > > definition means no SMN1 gene) and also has

> > >

> > > no SMN2

> > >

> > > > > > copies would not

> > > > > > be able to survive. If you have SMA Type 2,

> > >

> > > you must

> > >

> > > > > > have some SMN

> > > > > > protein, therefore you must have at least one

> > >

> > > copy

> > >

> > > > > > of SMN2, therefore

> > > > > > VPA might help.

> > > > > >

> > > > > > This article about VPA in Type 3 adults

> > >

> > > mentions the

> > >

> > > > > > SMN2 issue. It

> > > > > > might be true that the more SMN2 copies you

> > >

> > > have,

> > >

> > > > > > the more VPA will

> > > > > > increase the protein, but even a small

> > >

> > > increase may

> > >

> > > > > > be beneficial for

> > > > > > someone with very little to start with.

> > > > > > http://tinyurl.com/zol7t

> > > > > >

> > > > > > Good luck!

> > > > > >

> >

> > __________________________________________________

> >