On Sept. 29, part of my life was lost. My dog, Gus, who had been a staple of the Miller family household for 13 years, left the world. To where, we can only guess, but he left parts of his odd personality with us and gave us memories we will…
Edasalonexent, a potential oral therapy for Duchenne muscular dystrophy (DMD), can prevent the development of heart disease and help to preserve bone health, according to data from two studies in mouse models of DMD. Catabasis Pharmaceuticals’ investigational treatment was also well-tolerated when given for up to…
Santhera Pharmaceuticals has decided to stop SIDEROS, its Phase 3 trial assessing the effectiveness of Puldysa (idebenone) at delaying respiratory decline in patients with Duchenne muscular dystrophy (DMD) on a stable glucocorticoid dose. An interim data analysis of the study (NCT02814019), which was fully enrolled, determined that…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to PF-06939926, Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD). This designation is given to treatments that show considerable potential in addressing serious conditions for which available treatments fall short. It is…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, Solid Biosciences’ investigational gene therapy for people with Duchenne muscular dystrophy (DMD), the company announced. Updated safety and effectiveness data for all patients dosed to date,…
I have clear memories of listening to my fourth-grade friends talk about what their “boyfriends” got them for Valentine’s Day, or whispering between one another during class about who liked whom. My hope of one day having a stereotypical teenage romance carried into high school, but dwindled as…
As a journalist and person with Duchenne muscular dystrophy, I have a dual purpose. I want to inform people and hold those in power accountable. I also want to show that having a disability doesn’t detract from my career goals. U.S. National Disability Employment…
A single dose of SRP-9001, Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy (DMD), continues to be safe and to improve motor function in four boys treated two years ago, according to data from an ongoing Phase 1/2 trial. Updated results from Study-101 (…
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