Assertion: muscular dystrophy | /r/HasProperty | ataxia
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text: But many challenges — from the likelihood of no “one and done” gene therapy for diseases like muscular dystrophy, Pompe or Friedreich’s ataxia, to the sheer amount of vector needed for both systemwide and central nervous system treatment — remain, especially for rare neuromuscular ills, said Sharon Hesterlee, PhD, an executive vice president and chief research officer for the Muscular Dystrophy Association.