rdx asked in Science & MathematicsMedicine · 1 decade ago

PTC 124 Question?

today i saw it on a newspaper that PTC Therapeutics has been found a cure for Duchenne muscular dystrophy called PTC 124..i wonder this so called "cure" could be used on congenital dystrophy?

1 Answer

  • 1 decade ago
    Favorite Answer

    PTC 124 is for Duchenne muscular dystrophy and not for congenital muscular dystrophy. The proteins necessary for muscles affected by the genetic mutations in both muscular dystrophies are different. In Duchenne muscular dystrophy, dystrophin is affected while in congenital muscular dystrophy the mutations afflicting the muscles are on other proteins. Read more: http://www.emedicine.com/neuro/topic549.htm

    PTC 124 is used to halt the process causing Duchenne muscular dystrophy.

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