Up to 45% of patients with the most common inherited neuromuscular disease could benefit from a new "cocktail" drug being developed at the University of Alberta, according to research published this week in the Proceedings ...
Feb 23, 2022
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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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Researchers from North Carolina State University and the National Institutes of Health (NIH) have developed a method that stops allergic reactions by removing a key receptor from mast cells and basophils. Their work has implications ...
Nov 21, 2016
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The powerful US Food and Drug Administration (FDA) has given the green light to a drug developed by WA researchers Sue Fletcher and Steve Wilton for treating Duchenne muscular dystrophy.
Sep 21, 2016
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An RNA editing technique called "exon skipping" has shown preliminary success in treating a rare and severe form of muscular dystrophy that currently has no treatment, based on a new study from Northwestern Medicine and the ...
Oct 12, 2015
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