Last week, my girlfriend and I caught the 2020 filmed version of Lin-Manuel Miranda’s award-winning Broadway production “Hamilton.” The recording, which we watched on Disney+, featured the original cast performing at the Richard Rodgers Theatre in New York City. Exactly a month ago, we attended a live performance of the…
After nearly a year of treatment, Viltepso (viltolarsen) was well tolerated and tended to increase how fast boys with Duchenne muscular dystrophy (DMD) could stand from a lying position, but not significantly more than a placebo, according to a preliminary analysis of a Phase 3 study. The main…
My oldest son, Max, 18, graduated from high school last Sunday. Graduation is a milestone for all who achieve it, but since Max lives with Duchenne muscular dystrophy (DMD), it felt like an even greater accomplishment. I was bursting with emotion, including joy, relief, happiness, and fear.
Duchenne muscular dystrophy (DMD) can make it harder to engage in social activities, and patients often experience grief and frustration over lost function. Family, however, can be an invaluable source of support to help overcome the challenges of the disease. That’s according to a study, “‘You…
DYNE-101 and DYNE-251, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, continue to work as intended and may lead to functional improvements for patients, according to new clinical trial data. These findings come from ongoing Phase 1/2 studies, which may still be recruiting. DELIVER (NCT05524883), launched…
Findings from a Phase 2 trial testing losmapimod in adults with facioscapulohumeral muscular dystrophy (FSHD) have offered new insights into the best outcome measures for future clinical trials, according to final published data from the clinical study, which wrapped up in 2021. The Fulcrum Therapeutics treatment, as…
Regular readers of my column will know that my wife, Wendy, and I moved from Shanksville, Pennsylvania, to Pittsburgh in January 2023 to be closer to our children and grandchildren. There have been some issues, but the move has worked out well. We see a lot more…
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran (del-desiran, previously called AOC 1001), an investigational treatment for myotonic dystrophy type 1 (DM1) that’s about to enter Phase 3 clinical testing. The FDA gives the designation to experimental therapies that have the…
Life with Duchenne muscular dystrophy (DMD) doesn’t get easier as time goes by, but it’s made better by laughter, love, and everything that’s learned along the way. Three of my seven children have DMD: Max, 18; Rowen, 15; and Charlie, 13. As you might imagine, my life is…
Dosing has been paused in a Phase 3 trial testing fordadistrogene movaparvovec in children with Duchenne muscular dystrophy (DMD) following the sudden death of a boy who had been previously treated with the gene therapy, its developer Pfizer announced in a letter to the community. Pfizer indicated…
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