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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 Ultragenyx gene therapy for glycogen storage disorder; Overton faces confirmation hurdlesThe U.S. Food and Drug Administration has granted approval to Ultragenyx's gene therapy aimed at treating a glycogen storage disorder. This marks a regulatory milestone for the company and the rare…