The investigational gene therapy GALGT2 for Duchenne muscular dystrophy (DMD) was found to be safe in a preclinical study conducted at the Research Institute at the Nationwide Children’s Hospital, the researchers said. The study was funded by a $100,000 grant awarded by Parent Project Muscular…
Preliminary results of a Phase 1/2 study into the gene therapy candidate SGT-001 in Duchenne muscular dystrophy (DMD) patients show low levels of microdystrophin in patients’ muscles after three months of treatment with an initial low dose and continued safety, Solid Bioscience, the therapy’s developer, reported. Continued safety was also evident,…
Duchenne UK announced that it has awarded a sizable grant to Evox Therapeutics to advance in early testing a way of using gene therapy to deliver — without a virus as a transport agent — the dystrophin gene or shorter versions of it to people with Duchenne muscular dystrophy. The group’s…
The Muscular Dystrophy Association (MDA) has awarded a total of $9.9 million to 34 pioneering and innovative projects worldwide in its continuing efforts to support groundbreaking research for more than 40 neuromuscular diseases. “This latest round of grants is impressive in terms of total funding, number of grants awarded…
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