Sarepta Therapeutics SRPT
HealthcareMarket cap $10B1 connection
Sarepta develops gene therapies for Duchenne muscular dystrophy - the most common fatal genetic disease in children. Its Elevidys exon-skipping treatment gave hope to thousands of families. Despite regulatory hurdles, Sarepta is the leader in a disease that otherwise has no cure.
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- CRISPR TherapeuticsHealthcare · $4BCompeting DMD gene therapy approaches
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