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- Capricor’s Duchenne drug deramiocel receives negative FDA advisory vote on heart outcomeMindy Leffler, a parent of a young man with Duchenne muscular dystrophy, writes an opinion piece about Capricor’s phase 3 study of its drug deramiocel. She describes her son’s experience living ind…
- FDA approves Regeneron’s garetosmab for ultra-rare bone-forming diseaseThe FDA has granted approval to Regeneron's antibody garetosmab, which targets activin A, for the treatment of fibrodysplasia ossificans progressiva (FOP). FOP is an ultra‑rare disorder that causes…