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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Approximately 5,000 people in the U.S. develop amyotrophic lateral sclerosis (ALS) each year. On average, they survive for only two to five years after being diagnosed, according to the Centers for Disease Control and Prevention.
Nov 8, 2024
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Harvard stem cell biologists have pioneered a groundbreaking 3D organoid culture method for generating large numbers of adult skeletal muscle satellite cells, also known as muscle stem cells, in vitro.
Sep 12, 2024
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As a wave of gene therapies with multimillion-dollar price tags hit the market, many employers are dropping coverage and leaving families in a bind.
Sep 9, 2024
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Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, scientists at DZNE and the ...
Jul 26, 2024
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An international study has discovered that people living with the neurodegenerative condition spinal muscular atrophy (SMA) are at higher risk of developing fatty liver disease, suggesting that SMA patients may face additional ...
Jul 17, 2024
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In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or ...
Jun 25, 2024
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Spinal muscular atrophy (SMA) is a terrible disease in which a genetic mutation causes certain nerves responsible for sending signals to muscles to degenerate. This leads to muscles wasting away, and many patients have died ...
May 15, 2024
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