[echoes long ago]

just saw this and am passing this on for our members with CMT

http://www.stockmarketwire.com/artic...MT1A-drug.html

[Kitt]

Originally Posted by echoes long ago

just saw this and am passing this on for our members with CMT

http://www.stockmarketwire.com/artic...MT1A-drug.html

I read about it on another site. It is for CMT1A. It's the rat model thing and not tried on humans yet. It says it's comparable to Baclofen and when I looked that up it has so many serious side effects, etc. I would not want to be on that either. Of course medications do have side effects and not everyone gets them. But I would not want to be taking Baclofen either.

http://www.drugs.com/baclofen.html

We shall see how this all comes out. And it is for those CMTers who have CMT1A and it has not had human trials yet. It potentially lower toxic overexpression of PMP22 and potentially delay the progression of CMT1A. Lots of "potentially" and "could". I would not want to be in human trials for it. Just me.

[mrsD]

This looks very promising. Thanks for putting up the link Echoes!

Baclofen is not the issue here. It is the process by which the baclofen modifies that is useful, and being compared to. The mention of baclofen
is a "comparison"....Drug studies that do that are typically more accurate than those done against placebos.

There is no evidence at this time that the new product would have any of the same side effects as baclofen. It will be a different drug with its own characteristics. It might be more difficult or less depending on the human trials.

I just saw a new commerical on TV this lunchtime, watching CNN.
It was by the MDA association. (muscular dystrophy). It claimed
many new drugs coming for muscle and nerve treatment within the upcoming 5 years. Perhaps this is one of those?

[Kitt]

Originally Posted by mrsD

This looks very promising. Thanks for putting up the link Echoes!

Baclofen is not the issue here. It is the process by which the baclofen modifies that is useful, and being compared to. The mention of baclofen
is a "comparison"....Drug studies that do that are typically more accurate than those done against placebos.

There is no evidence at this time that the new product would have any of the same side effects as baclofen. It will be a different drug with its own characteristics. It might be more difficult or less depending on the human trials.

I just saw a new commerical on TV this lunchtime, watching CNN.
It was by the MDA association. (muscular dystrophy). It claimed
many new drugs coming for muscle and nerve treatment within the upcoming 5 years. Perhaps this is one of those?

What you say about Baclofen is probably true. We really do not know. And we do not know if there will be human trials or not. At any rate, this probably has a long way to go. It might be just the thing and we won't know until we hear further. Again, it is for CMT1A.

As far as the MDA association and new drugs for muscle and nerve treatment within the upcoming 5 years - again we do not know if it would be for CMT1A. I will be seeing this on their site and in their magazine I get. There are many types of Muscular Dystrophy and Charcot Marie Tooth disease is not one of them. CMT is of the peripheral nervous system (PNS) and Muscular Dystrophy is of the central nervous sytem (CNS). People who have MS are born with diseased muscles. CMTer's are born with healthy muscles. Again, CMT is not any type of MS. So time will tell.

[Susanne C.]

I know it is confusing that CMT is under the MDA family of diseases, although it is an atrophy not dystrophy of the muscles, but I would be happy for any treatment they develop. I don't really expect anything to change my life, but with at least one of my sons definitely affected and another showing symptoms I would love it if theirs could be arrested. People are being discouraged from having children by genetic counselors which is irrational since so many cases are late or mild onset.
We have CMT type 2, did not test for the subtype if any.

[Kitt]

Originally Posted by Susanne C.

I know it is confusing that CMT is under the MDA family of diseases, although it is an atrophy not dystrophy of the muscles, but I would be happy for any treatment they develop. I don't really expect anything to change my life, but with at least one of my sons definitely affected and another showing symptoms I would love it if theirs could be arrested. People are being discouraged from having children by genetic counselors which is irrational since so many cases are late or mild onset.
We have CMT type 2, did not test for the subtype if any.

Hi, Susanne,

I thought that you had CMT2 in your family. There is research being done for CMT2 but not certain which of the sub types. There are sixteen (16). And also research being done on other types of CMT. It would be nice to have a treatment available. We were told by an expert neurologist in CMT that if there was a cure/treatment it might leave you right where you are as far as symptoms. I wonder though how much a drug would cost and if one could afford it let alone if insurance would cover it. It does take years and years for a drug to be approved because big pharma is not going to take on orphan diseases like CMT is considered. In other words, there wouldn't be a great many people out there with it like there are with other diseases. We shall see.

Also, I do believe it is only you and I with a type of CMT who are on here. There have been a couple others but they have left quite some time ago. It would be nice to have a heading like the other group previous to this had. There it was plain as day - Charcot-Marie-Tooth. But there were few on there. So I'm sure that's why there isn't such a thing on this site. Anyone coming on would really have to dig to find anything on CMT under PN. Most CMTers are on other forums which are just for CMT. So there you go.

The research being done which was posted by Echos is in Germany. Interesting to see what the side effects might be if it ever comes to human trials. I would guess there would be some.

I would really like to know what the MDA is working on. I have some sources so hopefully will find out. It's a possibility that it is for people with Muscular Dystrophy and there are many types of that.

[Susanne C.]

I know that we are the only regulars on the site with CMT (yes, we do have type 2 which is rarer and gets less attention than 1a from researchers), but a lot of people have come and gone in the time I have been here who gave reason to suspect they had CMT. Idiopathic diagnosis, weakness and fatigue, hammertoes, restless legs, awkward gait, and oh mom had trouble with her feet. I think it is important to suggest to those people that it might be CMT because many of them will never hear it from their doctor or connect grandpa and mom and auntie's leg troubles with what they are experiencing. Some people's parents suffer in silence, and others, like my mother, never stop complaining about what were minor aches and pains.
I didn't even connect the fact that my half sister had leg braces and my aunt had to do "exercises" and my bio father couldn't get out of a chair and drank all day to numb the pain with this for years because they weren't "family", just a shameful secret.
It is worth keeping CMT out there as a possibility for the people it fits. While it isn't a treatable diagnosis, and is one of the least positive ones it is better to have a hard diagnosis than to keep pursuing answers that aren't there or worse to think it is in your head when it is really in your genes.

[Kitt]

Originally Posted by Susanne C.

I know that we are the only regulars on the site with CMT (yes, we do have type 2 which is rarer and gets less attention than 1a from researchers)

Yes, CMT2's depending on the type are rarer than CMT1A. However, they are doing research on some of the CMT2's as well as the other subtypes of CMT1. And some of the other types of CMT as well are being researched. I believe one reason that CMT1A gets a lot of the research is because it is one of the most common types of the disease and therefore more CMT people have it than the other types.

I do agree with you that there are people who come on here who very possibly do have a type of CMT. But it's never confirmed. The diagnosis is usually just idiopathic, or some such thing, which means the doctors don't know what they have. These people probably need further testing, expensive, look at their family history, and see a neurologist who might know something.

Thanks for your post. I do agree with your last statement as well.