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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Results of a compassionate-use study released in Stem Cells Translational Medicine show promising results for treating muscular dystrophies with mesenchymal stem cells (MSCs) derived from Wharton's jelly (WJ), a substance ...
Jul 27, 2021
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Roughly 1 out of 15,000 people born suffer from Facioscapulohumeral Muscular Dystrophy (FSHD), a rare disease that starts with the progressive weakening of facial muscles and then the shoulders and upper body. By the time ...
Jul 20, 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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A research collaboration based in Kumamoto University, Japan has discovered that muscles and the resident stem cells (satellite cells) responsible for muscle regeneration retain memory of their location in the body. This ...
Jul 6, 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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A novel gene editing technique developed by researchers at The Ohio State University Wexner Medical Center and College of Medicine is showing promise in correcting genetic mutations in Duchenne muscular dystrophy (DMD) and ...
Jun 25, 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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Getting in shape isn't easy. But after all that hard work, how long do we actually maintain it? Turns out that even the great effort we put into training, taking a bit of time off can mean that we become "unfit" much faster ...
May 14, 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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