dystrophin

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

BridgeBio has vaulted over the success bar it set for the trial, linking the candidate to significant improvements on biomarker and clinical outcomes.

Nick Paul Taylor Oct 27, 2025 7:00am

green light go go ahead advance yes streetlight

FDA advisers narrowly support Sarepta's DMD gene therapy

May 12, 2023 7:09pm

Sarepta sees muscle gains with DMD gene therapy

Oct 4, 2018 11:00am