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Will FDA's Accelerated Approval Pathway Facilitate Access to Gene Therapies as Applications Roll In?
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Industry observers are closely tracking how Sarepta navigates the agency's accelerated approval framework for its Duchenne muscular dystrophy gene therapy.
IU School of Medicine Team Nabs $3.8M NIH Grant to Study Muscular Disorder AAV Gene Therapy Safety
Researchers will study the body's immune response to muscle-directed gene therapies that can lead to immunotoxicities.