Author Archives: Vijaya Iyer, PhD

Gene Therapy SGT-001 Shows Signs of Microdystrophin Production in Muscles of Boys in IGNITE DMD Trial

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,…

MDA Awards $9.9M to 34 Projects Targeting Neuromuscular Diseases

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…