MeSH tree: C10.668.467.500
The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in the survival motor neuron ...
Dec 8, 2025
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A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature Medicine. The results of this ...
Dec 8, 2025
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Swiss pharmaceutical giant Novartis announced Sunday it had agreed to buy Avidity Biosciences, valuing the US biopharmaceutical company at $12 billion.
Oct 27, 2025
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Spinal muscular atrophy affects all the body's muscles. For a long time, it was considered a disease caused solely by the loss of nerve cells in the spinal cord. Now, a research team at Leipzig University's Carl Ludwig Institute ...
Oct 2, 2025
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Every movement we make, whether walking, speaking or even breathing, depends on the health of our muscles and nerves.
Sep 19, 2025
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For patients with nonambulatory type 2 or type 3 spinal muscular atrophy, apitegromab, a monoclonal antibody that inhibits myostatin activation, improves motor function versus placebo, according to a study published online ...
Aug 25, 2025
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From Johns Hopkins University School of Medicine, Charlotte J. Sumner, M.D., presents an editorial on a study by Richard S. Finkel and colleagues, who report an open-label, Phase II trial of the pre-messenger RNA splicing ...
Spinal muscular atrophy (SMA) is a rare genetic condition that causes progressive muscle weakness, which, when untreated, prevents infants with the most severe form from gaining motor development—never gaining the ability ...
Aug 14, 2025
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Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can lead to severe physical ...
Aug 11, 2025
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Ancient bacteria that have evolved to become integral to our cells—converting nutrients from food into energy—may also contribute to neurologic disorders, such as spinal muscular atrophy (SMA) and autism, according to research ...
Jul 24, 2025
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