Tag: ACVR1
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Regeneron Wins FDA Approval for Pasatru in Ultra-Rare Bone Disease
The FDA approved Regeneron’s Pasatru for adults with fibrodysplasia ossificans progressiva after Phase 3 data showed a 90–94% reduction in new heterotopic ossification lesions versus placebo.
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How Pasatru Blocks Abnormal Bone Formation in FOP
This BP Science explainer examines how mutant ACVR1 allows Activin A to drive abnormal SMAD1/5/8 signaling in FOP, and how Pasatru and Sohonos intervene at different points in the pathway.