Over 15 years ago, researchers linked a defect in a gene called survival motor neuron -- or SMN -- with the fatal disease spinal muscular atrophy. Because SMN had a role in assembling the intracellular machinery that processes ...
Jun 21, 2012
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A team of University of Missouri researchers has found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) – the leading genetic ...
Jul 16, 2012
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Spinal muscular atrophy (SMA) is a devastating pediatric neuromuscular disorder caused by loss-of-function mutations in the SMN1 gene, which prevents the body from producing enough of the survival motor neuron (SMN) protein ...
Dec 7, 2023
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A new treatment for Huntington's disease – a deadly brain disorder – has successfully completed first-in-human trials. The drug lowered levels of the harmful huntingtin protein in the spinal fluid of patients who took ...
Dec 14, 2017
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The enzyme Gemin3 was identified as the molecular 'bridge' between genes whose mutation or disruption causes amyotrophic lateral sclerosis (ALS), according to a new study in Nature's Scientific Reports from scientists at ...
Jan 16, 2020
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(HealthDay)—The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday.
May 28, 2019
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(HealthDay)—The U.S. Food and Drug Administration has approved the first oral, at-home drug for spinal muscular atrophy (SMA), the agency announced Friday.
Aug 11, 2020
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A University of Connecticut researcher is advancing the understanding of the devastating inherited condition known as spinal muscular atrophy.
Jan 29, 2013
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Spinal muscular atrophy is a debilitating neuromuscular disease that in its most severe form is the leading genetic cause of infant death. By experimenting with an ALS drug in two very different animal models, researchers ...
Apr 9, 2013
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If a disease affects motoneurons, cells that control voluntary muscle activity, researchers should focus their efforts on motoneurons to find potential treatments, right?
Aug 31, 2016
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