source
- Regeneron wins FDA approval for Pasatru, targeting rare bone disease FOPThe U.S. Food and Drug Administration has approved Regeneron Pharmaceuticals' drug Pasatru for the treatment of fibrodysplasia ossificans progressiva (FOP), a very rare condition in which muscle an…
- Experts call for modernizing rare disease evidence models using FDA plausible mechanism frameworkA recent paper in The Journal of Clinical Investigation argues that the evidentiary approach for rare diseases needs updating. The authors suggest applying the FDA’s plausible mechanism framework,…