Duchenne Surprise: Sarepta Prices Exondys 51 Below Expectations
It sparked at least one analyst to question how Sarepta came up with a price for FDA's first approved Duchenne muscular dystrophy treatment.
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The US FDA issued a complete response letter rejecting golodirsen based on risk of infection at intravenous infusion ports and preclinical renal toxicity.
Sarepta Commits To Rapid, Thorough Pivotal Study For DMD Gene Therapy Based On Functional Improvements
The company presented encouraging data on functional assessments of four boys treated with a micro-dystrophin gene therapy at the World Muscle Society, furthering momentum around the program.
Canaan Partners-backed biotech will investigate the therapeutic potential of three-way junctions in folded RNA. UC Berkeley and Duke sign separate license agreements related to CRISPR gene-editing technology.