Pancreatic ductal adenocarcinoma (PDAC) is the most lethal form of pancreas cancer. It's also the most common form of the disease. Potential treatments typically target a key mutated oncogene called KRAS. In some cases, PDAC ...
Jan 8, 2026
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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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