One of my favorite early-summer memories is from when my sons Max and Rowen were younger. Both lovely little boys — one blond-haired and blue-eyed, the other dark-haired and brown-eyed — squealed with laughter upon discovering a frog in our front yard. Max held the frog up in his hands…
SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to therapies that are designed to improve medical care for rare disorders, specifically defined…
Agamree (vamorolone) has been approved in the U.K. for treating Duchenne muscular dystrophy (DMD) in patients 4 and older. This approval by the Medicines and Healthcare products Regulatory Agency (MHRA) follows a similar decision in the European Union. In the U.S., Agamree is approved for patients…
There is joy after a diagnosis of Duchenne muscular dystrophy (DMD). There is joy after the loss of ambulation. There is joy in the midst of watching Duchenne slowly work its progressive, muscle-wasting way through my sons Max, 18, Rowen, 14, and Charlie, 12. That may be hard…
Race to End Duchenne, the signature fundraising program from Parent Project Muscular Dystrophy (PPMD), has presented Team Michael James with the 2024 “Racing for Every Future Team” award, while Michael Napoli was this year’s “Make Every Day Count” award recipient. Race to End Duchenne supports PPMD’s mission to cure…
As I look forward to the year ahead, I’m filled with a mix of emotions: unbridled joy at the thought of spending my first Valentine’s Day with my girlfriend, Amanda; a sense of dread as my caregiver Glenda leaves in July; and the euphoria of turning 29 in October. Writing…
Pennsylvania’s Somerset County, where I lived before moving to Pittsburgh a year ago, is situated in the Laurel Highlands, an area that is part of the Appalachian Mountain Range. At an elevation of more than 2,000 feet above sea level, the county often gets snow when other parts…
Dosing has begun in a Phase 2 clinical trial evaluating PepGen‘s PGN-EDO51 — a treatment candidate for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. The trial, called CONNECT1-EDO51 (NCT06079736), will evaluate the safety and preliminary efficacy of the experimental therapy in about 10 patients,…
When I stopped working at a job away from home in 2007, I never imagined it would be a permanent change. I was pregnant with my third child, Chance, who is now 16, and had decided that I wanted to stay home full-time with my kids. On my last day…
New trial data announced by Dyne Therapeutics show two experimental treatments for muscular dystrophy from the company’s FORCE platform appear to be working as intended in early clinical studies. Both DYNE-251 for Duchenne muscular dystrophy (DMD), being tested in a trial dubbed DELIVER, and DYNE-101 for…
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