Please provide your email address to receive an email when new articles are posted on . Ataluren preserved hand-to-mouth function in boys with nonsense mutation Duchenne muscular dystrophy. Ataluren ...
The FDA approved oral givinostat (Duvyzat) to treat Duchenne muscular dystrophy (DMD) in patients ages 6 years and older, the agency announced Thursday. Givinostat is a histone deacetylase (HDAC) ...
The U.S. Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...
Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...
The group of LMU scientist Eckhard Wolf has developed a porcine DMD model with a mutation, which mimics the hallmarks of the human disease but develops them in an accelerated mode. Molecular, ...
Mice with Duchenne muscular dystrophy mutations show improvement with gene editing. The DNA needs to encode three things: the gene for Cas9, which cuts DNA; RNA that guides Cas9 to the right place to ...
The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the risks and benefits of the drug The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the ...
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