Treatment with exon 53-skipping therapy WVE-N531 led to significant increases in muscle dystrophin levels along with signs of improved muscle health and regeneration in boys with Duchenne muscular dystrophy (DMD). That’s according to new six-month interim data from the Phase 1b/2a FORWARD-53 trial (NCT04906460), which also found…
Capricor Therapeutics will soon begin filing an application seeking U.S. approval of its cell therapy deramiocel for cardiomyopathy, a disease of the heart’s muscle, in people with Duchenne muscular dystrophy (DMD). Following recent meetings with the U.S. Food and Drug Administration (FDA), Capricor will file a rolling…
I’ve been disabled for over 50 years, and I spent over 30 years working with people with disabilities. The language surrounding disability has evolved in that time. For the most part, that’s a good thing. As an example, person-first language has become more common. It’s more appropriate to introduce someone…
The Muscular Dystrophy Association (MDA) is providing neuromuscular disease (NMD) patients and their families with the opportunity to meet experts and other people on similar journeys through its Engage Symposiums, free in-person events that include disease-specific sessions as well as talks focusing on NMDs in general. The association…
I believe that all of us who live with a chronic illness deal with a future full of unknowns. We all wrestle with uncertainty regarding quality of life from year to year, month to month, and even day to day. My chronic illness is limb-girdle muscular dystrophy. The…
I’m going gray. Wait, I should clarify that I’ve been coloring my hair since I was 25. It started coming in gray long before I became a mom to three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. But after hiding my natural color…
SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). The cell replacement therapy from Somite Therapeutics uses artificial intelligence (AI) and large, complex data sets to improve production of multiple human cell types.
Last Saturday, I had the honor of participating in the third Singapore Health Patient Advocate Connection event (SPACe) hosted by the SingHealth Patient Advocacy Network (SPAN). As a cast member of the incredible SPANtastic Theatre plenary skit team, I not only had the chance to perform, but also…
I’ve had a walker with wheels for a decade. I got it after back surgery in 2014 and used it for a month or so. As a blind person with facioscapulohumeral muscular dystrophy (FSHD), I found it much more difficult to maintain my orientation without direct touch. I was…
Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at improving upper limb function and other clinical measures in people with facioscapulohumeral muscular dystrophy (FSHD), according to top-line data from the REACH Phase 3 trial. Participants given losmapimod did see improvements over 48…
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