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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…
- BioMarin to acquire Alesta Therapeutics for $275 million to expand bone disease pipelineBioMarin Pharmaceutical announced it will purchase Alesta Therapeutics for an upfront payment of $275 million. Alesta is developing an oral small-molecule drug, ALE1, aimed at treating hypophospha…