Health & Medicine

STAT+: Pharmalittle: We're reading about a Regeneron rare disease drug, an Ultragenyx gene therapy, and more

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Regeneron Pharmaceuticals has received U.S. regulatory approval for Pasatru, a drug aimed at treating fibrodysplasia ossificans progressiva (FOP), a rare disease causing abnormal bone growth. Separately, Ultragenyx Pharmaceutical’s gene therapy Genglycos was approved by the FDA to treat glycogen storage disease type Ia (GSDIa), offering hope for patients with this metabolic disorder. These approvals highlight advancements in treating ultra-rare diseases and could set precedents for future regulatory pathways for similar therapies.

Read the full article at STAT News

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