Treatment with Viltepso (viltolarsen) helps preserve motor function in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, according to four-year data from an open-label extension study. “These data represent the longest clinical experience of an exon 53 skipping therapy for the treatment of Duchenne…
The Duchenne muscular dystrophy community is reaping the benefits of innovations in rare disease diagnosis, research, and care. That’s part of what I learned last week while training to cover rare diseases with the National Press Foundation (NPF). I was selected for the NPF’s Rare Disease…
Warning: This column contains spoilers for the Netflix musical film “tick, tick…BOOM!” In my last column, I wrote about my relationship with the concept of crip time, which addresses how time works differently in the lives of disabled people. Crip time has enabled me to accept who I am…
My family enjoys attending sports events and theater productions. There aren’t many things we like more than a nice meal out followed by a game or a play. However, seating for families with wheelchair users is rarely ideal. I can only speak about my family’s experiences.
EDG-5506, Edgewise Therapeutics’ experimental oral therapy, continues to safely reduce muscle damage and improve physical function in adults with Becker muscular dystrophy (BMD), according to six-month interim data from the Phase 1 ARCH trial. Treatment led to a marked reduction in the average levels of key markers of muscle…
The Muscular Dystrophy Association (MDA) will host the MDA Tribute Awards Oct. 27 in Boston to honor four people who have contributed to progress in muscular dystrophy research and care, and in lobbying efforts on behalf of those with neuromuscular disorders, particularly Duchenne muscular dystrophy (DMD). The awards…
I never thought that being diagnosed with a rare muscle condition called Duchenne muscular dystrophy would one day lead me to Budapest, Hungary, but life always surprises me. A few weeks ago, I jumped on a flight to the historic European city to speak at a Duchenne conference.
School and Duchenne muscular dystrophy (DMD) don’t always go hand in hand. But of course, they can — and when they do, it’s beautiful. But even then, I doubt it’s come about without an extraordinary amount of work from the parents, the school district, or both. This school year,…
The U.K.’s National Institute for Health and Care Excellence (NICE) has published new draft guidance that does not recommend Translarna (ataluren) be covered to treat Duchenne muscular dystrophy (DMD) caused by nonsense mutations. According to NICE, although there’s evidence Translarna can benefit people with DMD, it’s not worth…
Santhera Pharmaceuticals has asked the European Medicines Agency (EMA) to approve vamorolone to treat Duchenne muscular dystrophy (DMD). The agency’s assessments of new approval requests — in the form of marketing authorization applications — take up to 210 days, but can be longer if the Committee for Medicinal Products…
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