Diseases, Conditions, Syndromes

Lab-grown muscles reveal mysteries of rare muscle diseases

Biomedical engineers at Duke University have developed a new technique to better understand and test treatments for a group of extremely rare muscle disorders called dysferlinopathy or limb girdle muscular dystrophies 2B ...

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 ...