News

Benitec Biopharma has begun the first study to support the start of clinical trials of BB-301, a potential gene therapy for people with oculopharyngeal muscular dystrophy (OPMD). The BB-301 Tissue Transduction Study is the first of three studies to be conducted in large animals, with the overall…

Two magnetic resonance measures — MRI and magnetic resonance spectroscopy (MRS) — of leg muscles are quality biomarkers that help to predict clinical milestones in Duchenne muscular dystrophy (DMD), including loss of walking ability, and may serve as outcomes measures in clinical trials, a natural history study reported. Its…

NS Pharma has launched a support hub, called NS Support, for people in the U.S. with Duchenne muscular dystrophy (DMD) and their healthcare providers. NS Support will provide patients and clinicians with information on product availability and program enrollment. The hub can be reached by telephone at 833-677-8778, Monday…

Hansa Biopharma has granted an exclusive worldwide license to Sarepta Therapeutics to develop and promote imlifidase as a gene therapy pre-treatment for people with muscular dystrophy who would otherwise be unable to receive such therapy.

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will be available to eligible patients in the U.K. for another year via the renewal of its Early Access to Medicines Scheme (EAMS), the company announced. In agreeing to this renewal,…

Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) seeking approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 45 skipping. “The completion of our casimersen submission is an important milestone in our journey to advance treatments…

The COVID-19 pandemic has forced many to shelter at home, caused millions of layoffs, and tightened budgets. But for families affected by Duchenne muscular dystrophy (DMD), who front extra medical costs and must decide between working and potentially exposing their son to the virus, the pandemic…

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a label update for Translarna (ataluren) to enable its use in Duchenne muscular dystrophy (DMD) patients who lost their ability to walk. The committee has specifically voted to remove…

A Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy type 2i (LGMD2i), has dosed its first healthy volunteer, ML Bio Solutions announced. The company also announced that children and adults with a confirmed LGMD2i genetic diagnosis are invited to join a natural history…