New gene therapy for muscular dystrophy offers hope

A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those ...


Researchers pioneer noninvasive gene therapy for brain disorders

A new method developed by a research team led by Rice University's Jerzy Szablowski offers hope for treating brain disorders through gene therapy. The innovative approach could transform treatments for inherited conditions ...

Diseases, Conditions, Syndromes

FDA expands approval for Duchenne muscular dystrophy gene therapy

The U.S. Food and Drug Administration has expanded the approval of Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in individuals ≥4 ...

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