News

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

This year, the Muscular Dystrophy Association (MDA) will celebrate MDA National Muscular Dystrophy Awareness Month, held every September, with a series of online events. The events will be held virtually for the first time to ensure maximum participation, while promoting social distancing and other safety measures for people with…

An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping has been accepted and placed under priority review by the U.S. Food and Drug Administration (FDA), Sarepta Therapeutics announced. A regulatory decision is expected no later than Feb. 25, 2021. The…

NS Pharma‘s Viltepso (viltolarsen) is now commercially available in the U.S. for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. The FDA conditionally approved Viltepso earlier this month, and continued approval remains contingent upon confirmation of clinical benefit in the Phase 3 RACER53…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

A Phase 1 clinical trial of EPM-01 ((+)-epicatechin), a potential oral treatment of Becker muscular dystrophy that targets cellular energy, has enrolled its first patients. This one-year study of Epirium Bio‘s small molecule therapy is taking place at three U.S. locations: the University of California, Los Angeles, the University…

The LELANTOS Phase 3 clinical trial, which is evaluating FibroGen‘s pamrevlumab in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, has enrolled a first patient. This global study is currently recruiting up to 90 boys and men, ages 12 and older, using…

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…