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Gene Therapies Hold Promise for Neuromuscular and Other Rare Diseases
Challenges remain, however, to therapy development for a limited patient population.
United states
Fidlerb sarepta
Muscular dystrophy association conference
Hurdles to gene therapy development
National center
Translational sciences
Muscular dystrophy association
Advancing translational sciences
Therapy successes
Sarepta therapeutics
Gene therapy options
Gene therapy
Dystrophy association conference
Biologics evaluation
Advancing translational
Dystrophy association
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