Novartis acquiring US firm Avidity Biosciences for $12 bn
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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Disease or Syndrome: Spinal Muscular Atrophy
Medical news and research articles
Cerebellum found to contribute to symptoms in spinal muscular atrophy
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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Neuromuscular disease care is entering a new era of hope and innovation
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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Apitegromab improves motor function in spinal muscular atrophy
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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Open-label Phase II trial reports early motor milestones with risdiplam
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 ...
Clinical trial shows newborns with spinal muscular atrophy can start treatment at birth
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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What you should know about spinal muscular atrophy
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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Research brings hope for new treatments for spinal muscular atrophy, autism
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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A step forward in treating serious genetic disorders in utero
Evidence is mounting that clinicians can treat serious genetic disorders prenatally by injecting medicine into the amniotic fluid, thus preventing damage that begins in utero.
May 14, 2025
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Promising results from first prenatal therapy for spinal muscular atrophy
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in utero treatment of SMA with the orally administered ...
Feb 20, 2025
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