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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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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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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The U.S. Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).
Feb 18, 2025
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Three people with a muscle-destroying disease destined to worsen got a little stronger—able to stand and walk more easily—when an implanted device zapped their spinal cord.
Feb 8, 2025
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A new drug-free, minimally invasive intervention targets the root cause of progressive loss of neural function in spinal muscle atrophy (SMA), an inherited neuromuscular disease. An intervention, which involves electrical ...
Feb 5, 2025
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