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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…
- Leo Pharma to acquire experimental rare-skin disease drug dersimelagon in up to $435 million dealLeo Pharma announced it will acquire the rights to dersimelagon, an experimental oral therapy currently under FDA review, in a transaction that could total up to $435 million in upfront and near‑te…