Power chair use can feel like a burden. Most people I hang around don’t use a wheelchair so it makes me feel out of place. I also have to find alternate routes to get into places like restaurants and offices, which is inconvenient for all who accompany me. However,…
A new mouse model of Duchenne muscular dystrophy (DMD) can help researchers test potential gene editing therapies by showing real-time levels of the dystrophin protein in muscles and the heart, a study suggests. The new strategy is a sensitive,…
A glance around the walls of Barry J. Byrne’s office reveals a lot about the pediatric cardiologist who runs the Powell Gene Therapy Center at University of Florida (UF). In one corner is an unusual painting by 9-year-old Will Barkowsky of Jacksonville, Fla. Will, the first boy with…
CureDuchenne has begun taking blood samples and skin biopsies to facilitate research on new treatments for Duchenne muscular dystrophy. The CureDuchenne Biobank is a partnership involving neurologist Tahseen Mozaffar, MD, of the University of California-Irvine and RUCDR Infinite Biologics of Piscataway, New Jersey.
A new technique that compares RNA production between an individual’s two gene copies may help find genetic alterations that lead to muscular dystrophy and other rare diseases, a study suggests. The research, “Genetic regulatory variation in populations informs transcriptome analysis in rare disease,” appeared in the…
Long-term treatment with Puldysa (idebenone) provides a stable reduction of lung function decline by approximately 50% in people with Duchenne muscular dystrophy (DMD), a study shows. The research, “Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy,” was published in…
It’s over a month since my gallbladder surgery, and I couldn’t be happier with my healing process. I previously wrote about adjusting to life after gallbladder removal and mentioned using supplements to regulate my digestive system. In this column, I share a review of my experience with those supplements.
NS Pharma will share future plans for viltolarsen, its investigational exon-skipping therapy for Duchenne muscular dystrophy (DMD) in a webinar hosted by Parent Project Muscular Dystrophy (PPMD) on Wednesday. The company will discuss viltolarsen’s mechanism of action and its current regulatory status during the one-hour webinar,…
When I pray, I like to start by thanking God for all of the blessings he has bestowed on me. It reminds me of how much I have to be thankful for. We often forget to thank God, our friends, and our families, yet I think it’s important to express…
Parent Project Muscular Dystrophy (PPMD) announced it is updating its global registry with a new, user-friendly app intended to empower people with Duchenne muscular dystrophy and their families to become more informed and engaged. Created by Thread, a company that specializes in remote and decentralized patient…
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