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- FDA grants approval to Ultragenyx's Genglycos gene therapy for ultra-rare glycogen storage disorderUltragenyx announced that the U.S. Food and Drug Administration has approved its gene therapy called Genglycos. The therapy targets an ultra-rare glycogen storage disorder that can lead to severe…
- Capricor Therapeutics likely faces FDA rejection of its Duchenne cell therapyCapricor Therapeutics is working to avoid an imminent FDA rejection of its cell‑based treatment for Duchenne muscular dystrophy. The company says it remains confident in the drug, but the regulator…