Until recently, babies and children with spinal muscular atrophy (SMA) lacked any type of treatment capable of stopping the neuromuscular disease from progressing. In SMA, damaged motor neurons in the spinal cord fail to ...
Aug 20, 2021
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Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.
Aug 3, 2021
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Babies born with the rare, inherited motor neuron disease spinal muscular atrophy are, without treatment, unlikely to reach their second birthday. When, as a researcher in the 1990s, I became aware of the disease there were ...
Jul 20, 2021
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Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease. ...
Jun 29, 2021
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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.
Jun 23, 2021
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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 7, 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 6, 2021
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Scientists have identified a way to "rescue" muscle cells that have genetically mutated, paving the way to a possible new treatment for rare childhood illness such as Duchenne Muscular Dystrophy (DMD).
Feb 22, 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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