Biotechnology company
Appears in 2 stories
Marketing Elevidys under restricted label
For three years, Sarepta Therapeutics has been the only company in the world selling a gene therapy for Duchenne muscular dystrophy. Regenxbio's trial result on May 14, 2026, sets up a second contender for U.S. Food and Drug Administration (FDA) approval.
Updated 2 days ago
Incumbent leader facing safety setbacks
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 May 7
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