Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people.
Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people.
A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
SAR446268 is a one-time adeno-associated viral gene therapy that delivers a vectorized RNA interference to silence DMPK expression. The Food and Drug Administration (FDA) has granted Fast Track ...
(RTTNews) - Dyne Therapeutics Inc. (DYN), a clinical-stage company developing treatments for genetically driven neuromuscular diseases, is advancing its programs in Myotonic Dystrophy and Duchenne ...
Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast ...
A recent discovery of a molecular connection between autism and myotonic dystrophy, a type of neuromuscular disease, may provide a breakthrough on how clinicians approach autism spectrum disorder. The ...
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