Pictured: A boy in a wheelchair over a muscle texture background/Taylor Tieden for BioSpace
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.
And the momentum isn’t slowing down. The DMD pipeline is flush with investigational treatments making their way through mid- and late-stage clinical trials, with several companies expecting data readouts in 2024.
“Where we are this year has been built upon activity, investments and trials over the past two, three and four years,” Michael Kelly, chief scientific advisor at CureDuchenne, told BioSpace in an email. “I think this could end…
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