Researchers rescued mice from early death caused by a muscle-weakening disease, not by correcting the flawed gene that causes it, but instead by targeting another protein in the same signaling pathway.
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In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children's Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked ...
May 07, 2021
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In a mouse study, National Institutes of Health researchers have identified and mapped a diverse spectrum of motor neurons along the spinal cord. These neurons, which send and receive messages throughout the body, include ...
Apr 30, 2021
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The rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval. Investigators evaluated a newly approved drug, onasemnogene abeparvovec, ...
Apr 06, 2021
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Trinity researchers are studying how the brain re-wires itself in neurological disease. The team is building treatments for today's more common global conditions like motor neurone disease (MND/ALS) and spinal muscular ...
Nov 17, 2020
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Penn Medicine researchers have developed a new targeted approach to prevent a toxicity seen in the sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. It's an important hurdle to clear, ...
Nov 11, 2020
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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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The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in ...
Jul 02, 2020
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The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. ...
Jun 11, 2020
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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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