Researchers at Yale have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered 'undruggable.' ...
Sep 11, 2020
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Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. The disease often results in death before ...
Aug 27, 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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Although macrophages (cells involved in the detection and destruction of bacteria and other harmful organisms as well as dead cells) are classified as immune cells functioning in the activation and resolution of tissue inflammation, ...
Aug 10, 2020
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The devastating effects of Duchene muscular dystrophy (DMD) can be mitigated if detection takes place in early childhood. Sadly, early diagnosis is rare. Now, University RNA researchers have discovered a novel method that ...
Aug 4, 2020
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More than three years after a clinical trial was prematurely ended for failing to show progress in healing heart attack scars, a prominent peer-reviewed journal is publishing some surprising results showing that the heart ...
Aug 4, 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 2, 2020
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Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy, according to a new study published today in the Proceedings of the National Academy of Sciences.
Jun 30, 2020
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Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with ...
Jun 15, 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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