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FDA review re Sarepta 21/6/24 expanded gene therapy, page-5

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    Myotonia dystrophy............. Saw today in a Perth newspaper, called Perth Now, a story about 2 people raising money in the Perth to Rottnest swim for this form of muscular dystrophy. Apparently it affects both males and females and becomes very serious around age 50. Muscles waste away and sufferers lose their ability to eat, walk or talk.
    I have never heard of this form of muscular dystrophy (very limited medical knowledge).
    Will our ATL1102 be able to deal with this disease?
    Thanks for any response,
 
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