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…
A few unfortunate circumstances recently have left me mentally exhausted by the endless recurrence of ableism. It feels as though ableism lurks around every corner, waiting for the opportune moment to rain on my sunshiny parade. Arming myself with an advocate seems necessary for braving the outside…
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…
The world can be overwhelming thanks to the deadly coronavirus pandemic, especially for someone dealing with muscular dystrophy. On top of the daily struggles — lack of mobility, physical exhaustion, and steroid-related side effects — there’s also the worry of getting sick and feeling the virus’s financial…
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…
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