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The importance of Pfizer’s candidate has been heightened in the minds of the DMD community after the only FDA-approved gene therapy for DMD, Roche/Sarepta’s Elevidys (delandistrogene ...

Sarepta recently filed for approval to extend the label for Elevidys to include all ambulatory DMD patients, despite missing its primary objective in a phase 3 trial. Image. Phil Taylor.

Ifetroban improved heart function and reduced cardiac damage biomarkers in people with DMD in a Phase 2 clinical trial.