5 DMD Data Readouts to Watch in 2024

The March approval of ITF Therapeutics’ Duvyzat marked the third new FDA-approved treatment in 10 months for Duchenne muscular dystrophy (DMD), a genetic disorder that causes progressive muscle weakness and degeneration. In June 2023, the regulator greenlit Sarepta’s Elevidys as the first-ever gene therapy for DMD. That approval was followed by Santhera Pharmaceuticals’ novel corticosteroid Agamree in October.