Satellos Bioscience’s SAT-3247 was safe, well-tolerated, and showed initial signs of increasing muscle strength in five adults with Duchenne muscular dystrophy (DMD), the company said. “We believe the findings from this Phase 1b study support our plan to advance SAT-3247 into a placebo-controlled Phase 2 trial,” Satellos CEO…
It’s been a couple of weeks since my last column. I’ve missed writing them. I can never explain how therapeutic it is for me to share my life as a caregiver through writing. I have seven children: Lexi, 24, Max, 19, Chance, 18, Rowen, 16, Charlie, 14, Mary, 10, and…
An investigational gene therapy called GNT0004 has demonstrated sustained stabilization of motor function at up to two years of follow-up in two boys with Duchenne muscular dystrophy (DMD). The boys received what was determined to be the effective dose of GNT0004, which was developed by Genethon, during the initial…
As a Duchenne muscular dystrophy survivor, I’ve spent much of my life on the outside looking in, especially when it comes to public events like concerts and sporting events. My condition affects not only my ability to move but also my breathing. I rely on a BiPAP…
The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne muscular dystrophy (DMD) who were treated when they were 8 to 9 years old, according to new trial analyses. Boys with DMD at this age are typically expected to show…
In September 1995, I began my first teaching job at a New Hampshire public middle school instructing students about band instruments and music. At the time, I was 10 years into my diagnosis with Becker muscular dystrophy. I wouldn’t be rediagnosed — this time, correctly — with…
Note: This story was updated May 19, 2025, to clarify the requirements needed for Elevidys to transition to full approval. The Japanese Ministry of Health, Labour, and Welfare (MHLW) has conditionally approved the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat some cases of Duchenne muscular dystrophy (DMD). The…
If you’ve read any of my recent columns, you know that I’ve had a lot of health struggles of late. I’m back on the medical merry-go-round with physical therapy, occupational therapy, medical appointments, and tests that tie me up multiple days every week. I’m struggling to stand up from…
Parent Project Muscular Dystrophy (PPMD), a nonprofit dedicated to ending Duchenne muscular dystrophy (DMD), has published an updated edition of the PJ Nicholoff Steroid Protocol, marking the 10th anniversary of its first release. Called PJ’s Protocol, the guidance is designed to help healthcare providers manage corticosteroid use…
Solve FSH, a venture philanthropic organization, has invested $3 million to support the development of Armatus Bio‘s microRNA-based therapy ARM-201 as a treatment for facioscapulohumeral muscular dystrophy (FSHD). ARM-201 is designed to block the production of DUX4, the protein that is abnormally produced in people with FSHD, a…
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