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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 buy Alesta Therapeutics for $275M, adding rare bone disease drug to pipelineBioMarin announced Tuesday that it will acquire privately held Alesta Therapeutics, paying $275 million upfront with potential additional payments tied to milestones. The deal, expected to close b…