As I grow older with FSHD, I’m thinking about my legacy

I just turned 66. I’m not sure if it was my birthday, the calendar changing, or perhaps the fact that the men in my family don’t live especially long lives, but I’ve really been thinking about what kind of legacy I’ll leave behind when I pass from this world to…

Throughout 2023, Muscular Dystrophy News Today worked diligently to cover new scientific research, treatment developments, and clinical studies for muscular dystrophy (MD). Here are the 10 most-read stories of that year. No. 10 – With EMBARK trial data, Sarepta seeks to expand Elevidys’ approval Elevidys (delandistrogene moxeparvovec-roki) is a…

People with Becker muscular dystrophy (BMD) caused by specific mutations tend to be younger when they first start using a wheelchair, a new study reports. The study, “Natural history of Becker muscular dystrophy: a multicenter study of 225 patients,” also provides new insights into the frequency of…

Disease progression with Becker muscular dystrophy (BMD) varies considerably, and depends on a patient’s age — with progression largely seen only in adults — and type of mutation, a three-year study of BMD’s natural history shows. “We … provide evidence that decline/improvement in function would be hard to measure…

I forgot how scary hope can be. There was a time, a few short months, when I realized I was giving up. I accepted that Duchenne muscular dystrophy (DMD) would continue to progress in my sons, and there wasn’t much I could do about it. I hadn’t thrown in…

HuidaGene Therapeutics’ investigational gene-editing therapy, called HG302, for Duchenne muscular dystrophy (DMD) has been granted a rare pediatric drug designation by the U.S. Food and Drug Administration (FDA). This status is intended to incentivize companies to develop treatments for rare and serious or life-threatening diseases affecting people under…

On Dec. 10, I performed “Stand by Me” by Ben E. King at the year-end music recital hosted by ART:DIS, a Singapore organization that supports the artistic development of members of the disability community. I dedicated the song to my pillars of support in my life with Duchenne…

The European Commission has approved Agamree (vamorolone), a dissociative corticosteroid — a treatment expected to have fewer side effects than standard steroid medications — for patients ages 4 and older with Duchenne muscular dystrophy (DMD). The decision applies to all member states of the European Union, plus Iceland,…

“The stages of Duchenne.” I remember seeing this heading on many of the websites I obsessively read when I learned that my three sons, Max, 18, Rowen, 14, and Charlie, 12, had Duchenne muscular dystrophy (DMD). The stages of Duchenne are like a timeline of disease progression. Parent…

In October, the U.S. Food and Drug Administration (FDA) approved Agamree (vamorolone) for treating Duchenne muscular dystrophy (DMD) patients ages 2 and older, offering them a more tolerable, but similarly efficacious, oral corticosteroid option. Clinical trial evidence indicates Agamree might ease muscle damage and minimize the side effects…