Genetics

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 ...

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 ...

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

Experts develop immune-enhancing therapies to target tuberculosis

Experts are working on novel immune-enhancing therapies called host-directed therapies to use the body's own immune system to target tuberculosis, with hopes that they could tackle even the drug-resistant forms of the disease. ...

Genetics

Gene discovered that can protect against severe muscle disease

A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, or muscular dystrophies. This is shown in a new study carried out at Umeå University, Sweden, ...

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