Chief Executive Officer, Avidity Biosciences
Appears in 1 story
Preparing accelerated-approval filing for competing exon-44 drug
Boys born with Duchenne muscular dystrophy typically lose the ability to walk before their teens and rarely live past 30. The first drugs designed to slow that decline reached the market in 2016, but they restore less than 1% of normal dystrophin, the muscle protein patients are missing. A decade later, a new wave of biotech companies is trying to deliver far more of that protein into muscle cells using engineered molecular shuttles.
Updated 2 hours ago
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