I am always learning more about life with Duchenne

When three of my sons — Max, 18, Rowen, 15, and Charlie, 13 — were diagnosed with Duchenne muscular dystrophy between July 2010 and August 2011, my learning curve was practically a vertical line. When the diagnosing doctor first said “Duchenne,” it was the first time I had heard…

The U.S. Food and Drug Administration (FDA) has approved the first generic version of Emflaza (deflazacort) oral suspension for the treatment of people with Duchenne muscular dystrophy (DMD). Called deflazacort oral suspension, the medication was approved for DMD patients 5 and older, a slightly older population than the…

Over the past couple months, I’ve shared my decision to try physical therapy in an attempt to slow the progression of my facioscapulohumeral muscular dystrophy (FSHD). I also shared that I haven’t seen a lot of benefit from the physical therapy, at least in terms of regaining…

After nearly three months of treatment with pitolisant, adults with myotonic dystrophy type 1 (DM1) experienced a reduction of excessive daytime sleepiness (EDS) and fatigue. That’s according to data from the Phase 2 trial (NCT04886518), which is testing the safety and efficacy of Harmony Biosciences’ therapy in…

I feel relaxed — refreshed, even. We’ve had almost two weeks at home without any appointments or travel. The kids are complaining of boredom, but I’m letting boredom reign! It’s a beautiful but rare occurrence for my family of nine. My husband, Jason, and I have seven children: Lexi, 23,…

After three years of treatment with Capricor Therapeutics’ experimental cell therapy CAP-1002, people with Duchenne muscular dystrophy (DMD) continue to show benefits in arm and heart function, new data from the HOPE-2 open-label extension (OLE) study shows. Topline data from the Phase 3 HOPE-3 trial (NCT05126758), which…

Satellos Bioscience has established a clinical advisory board, with experts in drug development and genetic muscle disorders, to help propel SAT-3247, an oral therapy candidate for Duchenne muscular dystrophy (DMD). “The formation of this clinical advisory board marks a major development step for Satellos as we continue our…

What’s it like to have three children with Duchenne muscular dystrophy (DMD)? That’s the question I’m asked more than any other, as a mom and caregiver to three sons with DMD: Max, 18, Rowen, 15, and Charlie, 13. Of course, that question is only asked by parents or grandparents…

In a column I wrote last month, I talked about finally deciding to try some physical therapy, despite more than a little pessimism that it would be effective. Actually, I’ve been struggling with this decision for over a year. I finally followed through and completed a five-week…

An engineered transfer RNA (tRNA) molecule developed by hC Bioscience could be a fix for the protein that’s lacking in Duchenne muscular dystrophy (DMD), supporting the company’s ongoing efforts to identify a lead molecule that may treat the disease. In a study with a mouse model of DMD,…