Parent Project Muscular Dystrophy (PPMD), the leading U.S. non-profit driving Duchenne and Becker muscular dystrophy care, research, and advocacy efforts, with a group of more than 120 advocates, will ...
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...
In human biology, the protein dystrophin is a shining example of Joni Mitchell’s classic line, “you don’t know what you’ve got ’til it’s gone.” Dystrophin stabilizes muscle cells. In its absence, the ...
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First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy
Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...
Individuals diagnosed with Duchenne muscular dystrophy (DMD) face few treatment options. DMD is a progressive muscle weakness and degeneration with loss of contractibility caused by one of several ...
A study by Indiana University School of Medicine researchers sheds new light on the development and treatment of a rare form of muscular dystrophy. The study's findings were recently published in ...
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