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BridgeBio aces phase 3 rare disease test, clearing path to FDA

https://www.fiercebiotech.com/biotech/bridgebio-aces-phase-3-rare-disease-test-clearing-path-fda
1•randycupertino•2h ago

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randycupertino•2h ago
Exciting results for this rare genetic muscle-wasting disorder Limb-Girdle Muscular Dystrophy (LGMD2I/R9) trial, and sounds like the FDA is on board with the surrogate endpoint. Could be big validation for BridgeBio’s rare disease strategy.