News

An advisory committee of the U.S. Food and Drug Administration (FDA) has voted narrowly in favor of accelerated approval for SRP-9001 (delandistrogene moxeparvovec), an experimental gene therapy for Duchenne muscular dystrophy (DMD). While the FDA is not obligated to abide by the committee’s vote, the agency will consider…

An advisory committee of the U.S. Food and Drug Administration (FDA) will be meeting  Friday to discuss SRP-9001 (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD) that’s up for possible approval. The meeting, which will be livestreamed on YouTube, is scheduled from 9 a.m. to…

In people with Becker muscular dystrophy (BMD), both an elevated creatine-to-creatinine ratio and lower myostatin muscle protein levels in the bloodstream are associated with worse motor performance, a study reported. These two potential biomarkers predicted current functional abilities when combined with age but not disease progression over four years. Both…

After two years of treatment with losmapimod, people in a clinical trial with facioscapulohumeral muscular dystrophy (FSHD) showed no upper limb function worsening. Leo Wang, MD, PhD, of the University of Washington, presented the findings at the American Academy of Neurology (AAN) annual meeting in the talk, “…

Motor function improvements seen in boys with Duchenne muscular dystrophy (DMD) one year after being given the gene therapy SRP-9001 (delandistrogene moxeparvovec) continued for a second year, according to new clinical trial data presented at the recent American Academy of…

AOC 1044, an investigational exon 44-skipping therapy for Duchenne muscular dystrophy (DMD), has been granted fast track status by the U.S. Food and Drug Administration (FDA). The designation is intended to accelerate the therapy’s development and expedite its approval by providing more frequent meetings with the FDA and discussions…

IPS Heart has received rare pediatric drug designation for its GIVI-MPC and ISX9-CPC, two experimental single-course stem cell therapies for Duchenne muscular dystrophy (DMD). The designation is given by the U.S. Food and Drug Administration (FDA) to spur treatments for any rare disease that can be serious or…

Having reached an agreement with the U.S. Food and Drug Administration (FDA), NS Pharma will be launching a Phase 2 clinical trial to evaluate its investigational exon 44 skipping therapy for Duchenne muscular dystrophy (DMD). Trial details will be forthcoming once enrollment is imminent, according to NS Pharma,…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to RGX-202, a one-time gene therapy for Duchenne muscular dystrophy (DMD) that is in early clinical trials. The FDA gives this designation to investigational treatments that have the potential to address unmet clinical care needs for…

Levels of titin, a muscle protein, were found to be elevated in the urine of boys with Duchenne muscular dystrophy (DMD) — making it a potential, novel, non-invasive biomarker for the genetic disease, a study demonstrated. Using a standard DMD mouse model, a lack of dystrophin, the protein missing…