News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

The U.S. Food and Drug Administration (FDA) has conditionally approved NS Pharma‘s Viltepso (viltolarsen) for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping…

The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather feedback from those with Duchenne muscular dystrophy (DMD) who received Santhera Pharmaceuticals‘ investigational treatment Puldysa (idebenone), Duchenne UK announced in a press release. This treatment is designed to slow the…

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…