Kevin Schaefer, the associate director of community content at Bionews, the parent company of this website, recently shared a thought-provoking article from Psychology Today titled “How Chronic Illness and Masculinity Intersect.” It has me thinking about what kind of man I am and want to be. The…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD) that ultimately aims to slow the progression of the genetic condition. The FDA awards this status to investigational medicines that are designed to treat serious,…
I’m officially a mother-in-law! That’s a new title for me. I’m a mom to the seven children I share with my husband, Jason. I’m also a special needs mom and warrior mama to my three sons living with Duchenne muscular dystrophy (DMD). And now I’m also a mother-in-law…
The experimental gene therapy RGX-202 has been found to be well tolerated at a high dose, with biomarker data indicating it is working as designed to increase production of the microdystrophin protein in boys with Duchenne muscular dystrophy (DMD). That’s according to updated interim findings from the ongoing…
Starting last week, I’ve been trying to get my life back on track after enduring a few difficult months. As I recently shared, life has been challenging since I left my job in April. Then, in May, I faced an unexpected hospitalization, and June brought relationship tension. In July,…
A low dose of PGN-ED051, PepGen’s investigational exon 51-skipping therapy, safely increased dystrophin protein levels in people with Duchenne muscular dystrophy (DMD), according to early Phase 2 clinical trial data. The therapy’s effects either were comparable to or greater than what has been observed in studies of…
Muscular dystrophy entered my life in the fall of 1984. I was 11 years old and beginning my sixth year of school. Along with neighborhood friends, I would walk to and from my elementary school every day. That September, I remember feeling very tired on the short walk down…
Duchenne muscular dystrophy (DMD) touches every part of my family’s life. Jason, my husband of 23 years, and I have seven children. Three of them — Max, 18, Rowen, 15, and Charlie, 13 — live with DMD. We’re now in the middle of what could be one of…
Pfizer has discontinued development of fordadistrogene movaparvovec, its investigational gene therapy for Duchenne muscular dystrophy (DMD), after recent Phase 3 trial data indicated a failure to improve motor function in boys with the neuromuscular disease. Patients who have already received the gene therapy in the CIFFREO…
Duvyzat (givinostat), a newly approved treatment for Duchenne muscular dystrophy (DMD), is now available in the U.S., according to an announcement from ITF Therapeutics, which will be selling the therapy. “Following the FDA [Food and Drug Administration] approval of Duvyzat, our team has been focused on making…
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