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2) What does SCAD mean? ETF? ETF-DH?

3) CAT testing?

ETF...electron-transport function...and then the dh is probably dehydrogenase...I taking a stab at both of those but I think they are right given in context of the report.

CAT testing is just scans I think

deb

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> I recieved Dr. Cohen's report and reccomendations in the mail

today. I feel

> as if I've been slugged in the stomach. I was blown out of the

water by some of

> what he had to say. First off he spoke again of how I'm leaking a

huge amount

> of carnitine through my kidneys, because at max dose of IV Carnitor

my blood

> levels are only reaching low normal when they should be

astronomically high.

> He is questioning whether the carnitine is even getting back into

the muscle.

> Dr. Rhead reported from my fibroblasts testing that I have a mild

SCAD defect

> or defect in the electron transfer flavoprotein or its

dehydrongenase. Dr.

> Cohen was quick to say that he doesn't believe these results in

anyway exclude my

> working diagnosis of a carnitine transporter defect in my renal

tubes.

> Dr. Cohen then went on to make his reccomendations. 1)Continue

the IV

> Carnitine although I'm not improving. 2) Request Dr. Rhead to do

immunoblotting

> of SCAD, ETF and ETF-DH. 3) Discuss my case again with Dr. Hoppel

and discuss

> my case again with the whole CIDEM committee at their meeting this

week. 4)

> Strongly advises a muscle biopsy for a) pathology B) electron

transport chain

> enzymology on the muscle and mitochondria. c) acylcarnitine profile

on the

> muscle and mitochondria. d) CPT1, CPT2, and CAT testing of the

muscle and

> mitochondria. He said he realizes the risks involved with having

this biopsy and that

> it will require travel, general anesthesia and a surgery, but that

these would

> need to be balanced against the possible benefit. His closing

statement was

> and this will have to be 's final choice.

> I was shocked by his urging to have the muscle biospy because

it had

> never really talked about before. When he spoke with my mom a week

ago he didn't

> mention any idea of it all to her. I really appreciated how he

phrased his

> letter though and that he is leaving the decision up to me. I

realize I'm 19 yrs

> old now and legally it is my choice, but I thought that was very

nice. I'm

> going to pray alot about this and then decide what I should do. I

need your HELP

> though. I have no idea what those abbreviations mean and what all

this entails.

> I've heard all the horror stories about these biospsies but I also

know what

> help they can be as well.

> Specific questions if anyone can answer them please do. Thanks!

> 1) How would treatment change if we knew that the carnitine wasn't

getting

> into my muscle? What benefit to me is it to have this done other

than to have a

> name for what ails me?

>

> 2) What does SCAD mean? ETF? ETF-DH?

>

> 3) CAT testing?

>

> 4) How long should I stay afterwards? Even if they make me leave

the hospital

> I plan to stay at RMH because with not having anything major done

the last 2

> trips were just terribly debilitating.

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Might help if I write something before sending...

Of all the questions... I can answer only one...

What is SCAD? This information was obtained from NORD

www.rarediseases.org

Short Chain Acyl CoA Dehydrogenase Deficiency (SCAD) To purchase full-

text report ($7.50):

View Cart/Checkout

Copyright 1996, 1998

Synonyms of Short Chain Acyl CoA Dehydrogenase Deficiency (SCAD)

Acyl-CoA Dehydrogenase Deficiency, Short-Chain

Lipid-Storage Myopathy Associated with SCAD Deficiency

SCAD Deficiency

SCAD Deficiency, Adult-Onset (Localized)

SCAD Deficiency, Congenital (Generalized)

SCADH Deficiency

Disorder Subdivisions

General Discussion

Short-Chain Acyl-CoA Dehydrogenase Deficiency is an extremely rare

inherited disorder of fat metabolism belonging to a group of diseases

known as organic acidemias. Excessive amounts of acids and ammonia

accumulate in the blood and body tissues due to a deficiency of an

enzyme known as Short-Chain CoA Dehydrogenase. This enzyme is needed

to break down fatty acids in the blood.

There are two distinct types of Short-Chain Acyl-CoA Dehydrogenase

Deficiency. The more severe generalized form is present at birth

(congenital) and characterized by progressive muscle weakness

(hypotonia) and the accumulation of acidic waste product in the blood

(organic acidemia). The symptoms in infants associated with organic

acidemia may include poor feeding, frequent vomiting, and/or

lethargy. Congenital Short-Chain Acyl-CoA Dehydrogenase Deficiency

may lead to life-threatening complications. The milder adult onset

form of this disorder affects the skeletal muscles and is

characterized by muscle weakness.

I'm not too familiar with anything else... Could CAT testing be CAY

Scan?

This is all the infomation I have... And won't offer " advice " or

offer my " opinion " because it gets me into trouble :)

Dorothy

Mom blessed with 3 beautiful girls (Youngest with possible Mito)

Wife to ... My very best friend :)

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,

This may sound like I'm taking a HUGH thing and making it sound too simple and small, BUT I have one question. I have been mulling this over in my mind for the last day anyway and when I saw your letter I wondered if it's been there for you. Could it be the type of carnitine you're taking is not working well enough? I know Shenan tried the prescription Carnitor and it didn't seem to work for her so she switched right back to the Thorne brand she gets from our naturopath (can't remember if that's also a prescription but think it is even though she buys it directly from him).

I can't help but wonder if that could make a difference for you. If the one thing you need the most isn't getting where it needs to go, then of course, you'll keep having trouble.

Remember we were going to do the spiritual database and this is exactly what I meant. You asked, you prayed, and we can pray more, and then let's see what we think (feel inspired to do).

Love,

Dr. Cohen's report

I recieved Dr. Cohen's report and reccomendations in the mail today. I feel as if I've been slugged in the stomach. I was blown out of the water by some of what he had to say. First off he spoke again of how I'm leaking a huge amount of carnitine through my kidneys, because at max dose of IV Carnitor my blood levels are only reaching low normal when they should be astronomically high. He is questioning whether the carnitine is even getting back into the muscle. Dr. Rhead reported from my fibroblasts testing that I have a mild SCAD defect or defect in the electron transfer flavoprotein or its dehydrongenase. Dr. Cohen was quick to say that he doesn't believe these results in anyway exclude my working diagnosis of a carnitine transporter defect in my renal tubes.

Dr. Cohen then went on to make his reccomendations. 1)Continue the IV Carnitine although I'm not improving. 2) Request Dr. Rhead to do immunoblotting of SCAD, ETF and ETF-DH. 3) Discuss my case again with Dr. Hoppel and discuss my case again with the whole CIDEM committee at their meeting this week. 4) Strongly advises a muscle biopsy for a) pathology B) electron transport chain enzymology on the muscle and mitochondria. c) acylcarnitine profile on the muscle and mitochondria. d) CPT1, CPT2, and CAT testing of the muscle and mitochondria. He said he realizes the risks involved with having this biopsy and that it will require travel, general anesthesia and a surgery, but that these would need to be balanced against the possible benefit. His closing statement was and this will have to be 's final choice.

I was shocked by his urging to have the muscle biospy because it had never really talked about before. When he spoke with my mom a week ago he didn't mention any idea of it all to her. I really appreciated how he phrased his letter though and that he is leaving the decision up to me. I realize I'm 19 yrs old now and legally it is my choice, but I thought that was very nice. I'm going to pray alot about this and then decide what I should do. I need your HELP though. I have no idea what those abbreviations mean and what all this entails. I've heard all the horror stories about these biospsies but I also know what help they can be as well.

Specific questions if anyone can answer them please do. Thanks!

1) How would treatment change if we knew that the carnitine wasn't getting into my muscle? What benefit to me is it to have this done other than to have a name for what ails me?

2) What does SCAD mean? ETF? ETF-DH?

3) CAT testing?

4) How long should I stay afterwards? Even if they make me leave the hospital I plan to stay at RMH because with not having anything major done the last 2 trips were just terribly debilitating.

Please contact mito-owner with any problems or questions.

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,

ETF and ETF-DH would be the deffective enzymes that would cause the disease (SCAD, which is a FOD (fatty oxidation disorder).

Just like in my son who has GAII (FOD) the affected enzymes are called ETF-ETF-QO. The ETF-QO is the one affected and only partially functioning at 25%.

Not sure what the CAT testing means.

Hope this helps.

Also, if you'd like to know more, go to the FOD network and you can read about SCAD in more detail.

Krystena s

Caden 5 yrs GAII

Dr. Cohen's report

I recieved Dr. Cohen's report and reccomendations in the mail today. I feel as if I've been slugged in the stomach. I was blown out of the water by some of what he had to say. First off he spoke again of how I'm leaking a huge amount of carnitine through my kidneys, because at max dose of IV Carnitor my blood levels are only reaching low normal when they should be astronomically high. He is questioning whether the carnitine is even getting back into the muscle. Dr. Rhead reported from my fibroblasts testing that I have a mild SCAD defect or defect in the electron transfer flavoprotein or its dehydrongenase. Dr. Cohen was quick to say that he doesn't believe these results in anyway exclude my working diagnosis of a carnitine transporter defect in my renal tubes.

Dr. Cohen then went on to make his reccomendations. 1)Continue the IV Carnitine although I'm not improving. 2) Request Dr. Rhead to do immunoblotting of SCAD, ETF and ETF-DH. 3) Discuss my case again with Dr. Hoppel and discuss my case again with the whole CIDEM committee at their meeting this week. 4) Strongly advises a muscle biopsy for a) pathology B) electron transport chain enzymology on the muscle and mitochondria. c) acylcarnitine profile on the muscle and mitochondria. d) CPT1, CPT2, and CAT testing of the muscle and mitochondria. He said he realizes the risks involved with having this biopsy and that it will require travel, general anesthesia and a surgery, but that these would need to be balanced against the possible benefit. His closing statement was and this will have to be 's final choice.

I was shocked by his urging to have the muscle biospy because it had never really talked about before. When he spoke with my mom a week ago he didn't mention any idea of it all to her. I really appreciated how he phrased his letter though and that he is leaving the decision up to me. I realize I'm 19 yrs old now and legally it is my choice, but I thought that was very nice. I'm going to pray alot about this and then decide what I should do. I need your HELP though. I have no idea what those abbreviations mean and what all this entails. I've heard all the horror stories about these biospsies but I also know what help they can be as well.

Specific questions if anyone can answer them please do. Thanks!

1) How would treatment change if we knew that the carnitine wasn't getting into my muscle? What benefit to me is it to have this done other than to have a name for what ails me?

2) What does SCAD mean? ETF? ETF-DH?

3) CAT testing?

4) How long should I stay afterwards? Even if they make me leave the hospital I plan to stay at RMH because with not having anything major done the last 2 trips were just terribly debilitating.

Please contact mito-owner with any problems or questions.

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Hi! I am sure the results are very frustrating and still really tell you nothing. I can tell you that SCAD is a fatty oxidation disorder where you don't process fats efficiently. Most people do not have the full SCAD disease but have variants or mutations in the gene which is on exon 6. Some people have more than 1 mutation and experience muscle weakness, failure to thrive, possible low sugar, lack of desire to eat, vomiting and most important when my Zack gets sick, he gets hospitalized due to the lactic acid that builds up that can cause metabolic acidosis. He gets IV fluids to help flush the raised pH(acid) out of his body and keep him hydrated and sugar level up. Hope this helps. You can also go onto WWW.FODSUPPORT.ORG. This is a place where those that are affected w/ fatty oxidation disorders get support..much like this site.

Best Wishes,

Mom to Zachary 3 yrs old (SCAD Variant and Complex I and IV)

4 1/2 yrs old (not tested)

Re: Dr. Cohen's report

> I recieved Dr. Cohen's report and reccomendations in the mail today. I feel > as if I've been slugged in the stomach. I was blown out of the water by some of > what he had to say. First off he spoke again of how I'm leaking a huge amount > of carnitine through my kidneys, because at max dose of IV Carnitor my blood > levels are only reaching low normal when they should be astronomically high. > He is questioning whether the carnitine is even getting back into the muscle. > Dr. Rhead reported from my fibroblasts testing that I have a mild SCAD defect > or defect in the electron transfer flavoprotein or its dehydrongenase. Dr. > Cohen was quick to say that he doesn't believe these results in anyway exclude my > working diagnosis of a carnitine transporter defect in my renal tubes. > Dr. Cohen then went on to make his reccomendations. 1)Continue the IV > Carnitine although I'm not improving. 2) Request Dr. Rhead to do immunoblotting > of SCAD, ETF and ETF-DH. 3) Discuss my case again with Dr. Hoppel and discuss > my case again with the whole CIDEM committee at their meeting this week. 4) > Strongly advises a muscle biopsy for a) pathology B) electron transport chain > enzymology on the muscle and mitochondria. c) acylcarnitine profile on the > muscle and mitochondria. d) CPT1, CPT2, and CAT testing of the muscle and > mitochondria. He said he realizes the risks involved with having this biopsy and that > it will require travel, general anesthesia and a surgery, but that these would > need to be balanced against the possible benefit. His closing statement was > and this will have to be 's final choice. > I was shocked by his urging to have the muscle biospy because it had > never really talked about before. When he spoke with my mom a week ago he didn't > mention any idea of it all to her. I really appreciated how he phrased his > letter though and that he is leaving the decision up to me. I realize I'm 19 yrs > old now and legally it is my choice, but I thought that was very nice. I'm > going to pray alot about this and then decide what I should do. I need your HELP > though. I have no idea what those abbreviations mean and what all this entails. > I've heard all the horror stories about these biospsies but I also know what > help they can be as well. > Specific questions if anyone can answer them please do. Thanks!> 1) How would treatment change if we knew that the carnitine wasn't getting > into my muscle? What benefit to me is it to have this done other than to have a > name for what ails me? > > 2) What does SCAD mean? ETF? ETF-DH?> > 3) CAT testing?> > 4) How long should I stay afterwards? Even if they make me leave the hospital > I plan to stay at RMH because with not having anything major done the last 2 > trips were just terribly debilitating.Please contact mito-owner with any problems or questions.

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,

I cannot help out at all on the abbrevaitions.The pathology of

this disease is still way beyond me. I get lost lost on all of the

numbers and letters. I can comment on the muscle biopsy. I know when

we saw Cohen that the skin biopsy and blood work came first, but we

were told that a muscle biopsy may be helpful...then told it should

be done. I am sorry that this was not communicated to you and caught

you by surprise. I too have heard the horror stories surrounding this

procedure. had this done at the same time she had a liver

biopsy. There is a risk with anesthesia and nerve damage. I guess my

advice would be to figure out what could be gained by this, and weigh

it with how you are living now. Does the information they may gain

through this promise you hope for treatment, or is it only for the

knowledge. I know that you suffer from weakness and fatigue from this

monster of a disease. Maybe this could lead you in a new direction

with a better outcome. I know that 's health was so poor at

this time we felt we had to do this...one of our hail marys! You are

an adult and it is your decision. You may recieve a lot of opinions

here, including mine, but please don't feel pressure. It is your

decision and we will help and support you no matter what you decide.

Remember that I'm up here by Cleveland. I unfortunately can't help

with accomidations (no guest room)but I can travel to CCF to help

with support or stay with you while your parents take a break or two.

I am here for you!!Also remeber that this decision does not need to

be made today!

Dawn

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,

I know that you have been on a long journey that you are still traveling. I will pray that you will get some definitive answers that will help you feel better.

It sounds like you have been getting some good advice from this group.

You are my prayers.

Darlene

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Sorry to hear about the discouraging report. I won't offer any help with the diagnosis as I am in the dark as well with them. I think several people gave you good explanations. I will pray for you as I know how hard some of these decisions are. Let us know how God leads. Darla: mommy to Asenath Dr. Cohen's report I recieved Dr. Cohen's report and reccomendations in the mail today. I feel as if I've been slugged in the stomach. I was blown out of the water by some of what he had to say. First off he spoke again of how I'm leaking a huge amount of carnitine through my kidneys, because at max dose of IV Carnitor my blood levels are only reaching low normal when they should be astronomically high. He is questioning whether the carnitine is even getting back into the muscle. Dr. Rhead reported from my fibroblasts testing that I have a mild SCAD defect or defect in the electron transfer flavoprotein or its dehydrongenase. Dr. Cohen was quick to say that he doesn't believe these results in anyway exclude my working diagnosis of a carnitine transporter defect in my renal tubes. Dr. Cohen then went on to make his reccomendations. 1)Continue the IV Carnitine although I'm not improving. 2) Request Dr. Rhead to do immunoblotting of SCAD, ETF and ETF-DH. 3) Discuss my case again with Dr. Hoppel and discuss my case again with the whole CIDEM committee at their meeting this week. 4) Strongly advises a muscle biopsy for a) pathology B) electron transport chain enzymology on the muscle and mitochondria. c) acylcarnitine profile on the muscle and mitochondria. d) CPT1, CPT2, and CAT testing of the muscle and mitochondria. He said he realizes the risks involved with having this biopsy and that it will require travel, general anesthesia and a surgery, but that these would need to be balanced against the possible benefit. His closing statement was and this will have to be 's final choice. I was shocked by his urging to have the muscle biospy because it had never really talked about before. When he spoke with my mom a week ago he didn't mention any idea of it all to her. I really appreciated how he phrased his letter though and that he is leaving the decision up to me. I realize I'm 19 yrs old now and legally it is my choice, but I thought that was very nice. I'm going to pray alot about this and then decide what I should do. I need your HELP though. I have no idea what those abbreviations mean and what all this entails. I've heard all the horror stories about these biospsies but I also know what help they can be as well. Specific questions if anyone can answer them please do. Thanks! 1) How would treatment change if we knew that the carnitine wasn't getting into my muscle? What benefit to me is it to have this done other than to have a name for what ails me? 2) What does SCAD mean? ETF? ETF-DH? 3) CAT testing? 4) How long should I stay afterwards? Even if they make me leave the hospital I plan to stay at RMH because with not having anything major done the last 2 trips were just terribly debilitating. Please contact mito-owner with any problems or questions.

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,

SCAD is short-chain acyl-CoA dehydrogenase it is a genetic disorder that Cameron has two mutations on that gene. Unfortunately it is one of the more rare disorders and there is little info out there on it. Google has some and the FOD.org website has limited information and some others who have it. Cameron is also suspected to have SCAD secondary to and electron transport chain disorder which is in one of the five mito complexes. Usually, if SCAD is suspected it is a Complex II defect that involves transfer of flavoprotein or dehydrogenese. Cameron responds well to COQ10 and riboflavin (used for SCAD) and we were told that even if we had the muscle biopsy they would not do anything different in his treatment. His suspected dx is based on lab results and DNA mutations as well as skin fibroblasts which sound like they show similar results as yours. He did not have a carnatine deficiency at the time of testing. Hope that helps and is not too confusing. Also one of the tell tale signs of SCAD is elevated ethylmolonic acid that shows up in a urine organic acid test. Cam's was three times higher than the high end of normal.

-----Original Message-----From: LILQT4U1984@... Sent: Monday, July 14, 2003 10:17 AMTo: mito ; Subject: Dr. Cohen's report

I recieved Dr. Cohen's report and reccomendations in the mail today. I feel as if I've been slugged in the stomach. I was blown out of the water by some of what he had to say. First off he spoke again of how I'm leaking a huge amount of carnitine through my kidneys, because at max dose of IV Carnitor my blood levels are only reaching low normal when they should be astronomically high. He is questioning whether the carnitine is even getting back into the muscle. Dr. Rhead reported from my fibroblasts testing that I have a mild SCAD defect or defect in the electron transfer flavoprotein or its dehydrongenase. Dr. Cohen was quick to say that he doesn't believe these results in anyway exclude my working diagnosis of a carnitine transporter defect in my renal tubes.

Dr. Cohen then went on to make his reccomendations. 1)Continue the IV Carnitine although I'm not improving. 2) Request Dr. Rhead to do immunoblotting of SCAD, ETF and ETF-DH. 3) Discuss my case again with Dr. Hoppel and discuss my case again with the whole CIDEM committee at their meeting this week. 4) Strongly advises a muscle biopsy for a) pathology B) electron transport chain enzymology on the muscle and mitochondria. c) acylcarnitine profile on the muscle and mitochondria. d) CPT1, CPT2, and CAT testing of the muscle and mitochondria. He said he realizes the risks involved with having this biopsy and that it will require travel, general anesthesia and a surgery, but that these would need to be balanced against the possible benefit. His closing statement was and this will have to be 's final choice.

I was shocked by his urging to have the muscle biospy because it had never really talked about before. When he spoke with my mom a week ago he didn't mention any idea of it all to her. I really appreciated how he phrased his letter though and that he is leaving the decision up to me. I realize I'm 19 yrs old now and legally it is my choice, but I thought that was very nice. I'm going to pray alot about this and then decide what I should do. I need your HELP though. I have no idea what those abbreviations mean and what all this entails. I've heard all the horror stories about these biospsies but I also know what help they can be as well.

Specific questions if anyone can answer them please do. Thanks!

1) How would treatment change if we knew that the carnitine wasn't getting into my muscle? What benefit to me is it to have this done other than to have a name for what ails me?

2) What does SCAD mean? ETF? ETF-DH?

3) CAT testing?

4) How long should I stay afterwards? Even if they make me leave the hospital I plan to stay at RMH because with not having anything major done the last 2 trips were just terribly debilitating.

Please contact mito-owner with any problems or questions.

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Hi !

Thanks for your help. I would love to know the doctor's name.

Praying for you today.

shirley

On Thursday, July 17, 2003, at 04:19 AM, LILQT4U1984@... wrote:

> Thanks so much Shirley. Your prayers and support mean so much to me. I

> have a friend in California who I think saw Dr. Levine. I'm going to

> e/mail her today and double check because I know she was VERY pleased

> with who she saw. He was very compassionate and into researching

> mitochondrial diseases. He did alot of testing on her and said that if

> something came up in his research that he felt she needed to be seen

> again he would call her. Also said that if she had questions and needed

> to talk to him to just call his office and he would speak to her.

> Luckily my friend is doing better now and none of her labs indicated a

> mito problem. I'll let you know when I find out for sure. Have a good

> day.

>

>

> Please contact mito-owner with any problems or

> questions.

>

>

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