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

1 comments

• randycupertino 9 hours 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.