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- Ultragenyx secures first gene therapy approval for GSDIa as Sanfilippo asset decision pendingUltragenyx received accelerated FDA approval for its gene therapy DTX401, marketed as Genglycos, to treat glycogen storage disease type Ia (GSDIa). The approval comes as the company awaits a secon…
- 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…