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- Ultragenyx secures FDA approval for Glenglycos, first gene therapy for GSD IaUltragenyx announced FDA approval of Glenglycos, a one-time gene therapy for glycogen storage disease type Ia, a rare genetic disorder. The therapy delivers a functional gene to the liver to resto…
- 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…