News

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

The importance of communication and self-care for people with muscular dystrophy and other neuromuscular diseases who are pursuing their education during the COVID-19 pandemic was highlighted in a Facebook Live event hosted by the Muscular Dystrophy Association (MDA). The event featured…

The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial testing Solid Biosciences’ gene therapy SGT-001 for people with Duchenne muscular dystrophy (DMD), the company announced. In a letter to Solid, the FDA has requested more information about the gene therapy’s…

An investigational gene therapy for Duchenne muscular dystrophy (DMD), called SRP-9001 micro-dystrophin, was given fast track designation by the U.S. Food and Drug Administration (FDA) its developer, Sarepta Therapeutics, announced in a press release. Fast track status is given to therapies with the potential to…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vita Therapeutics‘ VTA-110, a candidate therapy for people with Duchenne muscular dystrophy (DMD). The designation grants various development incentives to medications that address rare disorders affecting fewer than 200,000 people annually in the…

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…