This first-in-human study of golodirsen showed its long-term safety and biologic activity in patients with Duchenne Muscular Dystrophy (DMD). The approved exon-skipping therapy is designed to enable the production of functional ...
Nov 30, 2021
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Muscular dystrophy describes a group of genetic diseases in which muscles progressively weaken and degenerate, with Duchenne muscular dystrophy (DMD) being the most common. There are no curative treatments for DMD, and available ...
Nov 29, 2021
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There is no consensus on when to introduce cardioprotective treatment in patients with Becker muscular dystrophy (BMD), and practice varies widely. A new study published in the Journal of Neuromuscular Diseases concludes ...
Sep 21, 2021
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Northwestern Medicine scientists have developed an antibody that they believe can be used to treat muscular dystrophy, findings that were published in Science Translational Medicine.
Sep 13, 2021
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The side effects of the Pfizer-BioNTech jab against COVID-19 are likely to be mild to moderate and clear up quickly in 12-15 year olds at high risk of complications from the infection because of certain coexisting conditions, ...
Aug 26, 2021
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Muscular dystrophy describes a family of congenital diseases that cause the progressive loss of muscle tissue. Severity varies across the family, but for many there is little in the way of treatment. In its newest study, ...
Aug 24, 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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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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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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