Quote:
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.
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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.