MAY 15, 2024 by Friedrich–Alexander University Erlangen–Nurnberg Credit: Med (2024). DOI: 10.1016/j.medj.2024.02.010Spinal 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 a painful death due to this rare condition. Genetic...
by Skolkovo Institute of Science and Technology Credit: Pixabay/CC0 Public Domain 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. Their results may lead to the development of drugs with less toxicity and fewer injections...
IOS PRESS Amsterdam, April 6, 2021 – 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, in a broader spectrum of patients in order to obtain expanded data on its side effects...
by Jonathan Gardner@ByJonGardner Jud Broadhurst was 14 years old when he began to notice the symptoms. Playing competitive soccer, Broadhurst would fall, seemingly “for no reason,” he said. But there was one, lurking in his genes. Broadhurst had a form of spinal muscular atrophy, a rare neuromuscular condition that was causing his muscles to weaken...
