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- Regeneron’s ultra-rare bone disorder drug receives U.S. approval after 30-year developmentRegeneron Pharmaceuticals announced that its therapy for an ultra-rare condition in which bone forms in soft tissue has been approved by the U.S. Food and Drug Administration. The disease, sometim…
- 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…