When I was 13 and fresh from spinal fusion surgery, I sat in a West End theater in London with my family, watching “Les Misérables.” The music stirred my soul and, for a fleeting moment, I imagined myself on stage. But that dream quickly faded. Accessibility barriers at school…
Preliminary data are expected this year for a Phase 1/2 clinical trial testing the investigational therapy SRP-1003 in people with myotonic dystrophy type 1 (DM1), according to an announcement from Sarepta Therapeutics. The trial recently hit one of two prespecified enrollment targets, prompting a review of safety data.
The steroid therapy Agamree (vamorolone), which is used for helping to preserve muscle function in people with Duchenne muscular dystrophy (DMD), will be sold and promoted in Turkey by Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. (GEN), per an agreement with Santhera Pharmaceuticals. “As leaders…
I’m a musician, and so are my brothers, Ron and Tim. We’ve all played professionally, but it’s been a long time since we’ve played together. That changed last month, but let me explain why it was such an event. Back in the late ’70s we were part of the…
A protein called MYOD, which is known to control the growth of muscle stem cells during muscle repair, has long been known to be able to turn on genes, but a recent study has now found that MYOD can also turn off certain genes. According to researchers, the findings may…
There is power in connection. As humans, we all learn this truth at a young age, starting with the people around us when we’re little: our family. As we grow, we connect with other kids at school, in church, and elsewhere in the community, and just by existing every day.
Enrollment is now ongoing in a first-in-human trial testing EPI-321, an epigenetic therapy designed to address the root cause of facioscapulohumeral muscular dystrophy (FSHD), a disease type in which symptoms like muscle weakness typically become apparent by adulthood. The Phase 1/2 clinical trial (NCT06907875) is expected to enroll…
As an artist with Duchenne muscular dystrophy (DMD), I’ve long recognized how creativity can foster meaningful connection, perhaps especially when rooted in design that’s accessible and inclusive. My partner, Amanda, and I recently spoke at GovTech’s Inclusive Design Week 2025 here in Singapore as part of the closing panel…
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Dyne Therapeutics’ DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. This designation is intended to accelerate the development and regulatory review of medications intended to treat serious…
I’m no longer a young mom, but I was once. In fact, I was a very young first-time mom, as my oldest daughter, Lexi, 24, was born when I was 21 years old. Six more children followed: Max, 19; Chance, 18; Rowen, 16; Charlie, 14; Mary, 10; and Callie, 3.
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