News

The Muscular Dystrophy Association (MDA) has launched the Gene Therapy Support Network (GTx) that will offer resources and guidance about approved gene therapies for people living with neuromuscular diseases. Its launch comes shortly ahead of a possible U.S. approval of SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics‘ experimental…

Avidity Biosciences is planning to double the dosage of AOC 1001 being given to a dozen people with myotonic dystrophy type 1 (DM1) who are receiving the experimental therapy in the MARINA-OLE clinical trial. The U.S. Food and Drug Administration (FDA) had placed a partial clinical hold…

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,…