Abstract
Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.
MeSH terms
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Clinical Trials as Topic
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Dystrophin / genetics
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Dystrophin / therapeutic use
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Exons / genetics*
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Genetic Therapy*
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Humans
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Muscular Dystrophies / genetics
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Muscular Dystrophies / therapy