Adaptive aids for Duchenne muscular dystrophy – Muscular …
Jun 21, 2024 · Assistive technologies. Advancements in technology have been a great help to people living with DMD, from enhancing accessibility with eye-tracking devices, smart …
NS Pharma DMD treatment gets FDA rare pediatric disease status
Sep 12, 2024 · NS-050/NCNP-03 is an antisense oligonucleotide, a short strand of genetic material. The therapy skips over exon 50, an approach amenable for about 4% of the DMD …
Sevasemten lowers markers of muscle damage in BMD: Trial
Dec 17, 2024 · “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of sevasemten,” Craig …
How I fully embrace life with limb-girdle muscular dystrophy
Sep 23, 2024 · At 40, I transitioned to a power wheelchair and purchased a van with a ramp and hand controls, which gave me the freedom to continue driving. Driving was a goal of mine, and …
Top 10 MD news stories of 2024
Jan 2, 2025 · Read the story here. No. 8 – PPMD conference highlighted DMD gene therapy and treatments. As part of our coverage of the 2024 Parent Project Muscular Dystrophy (PPMD) …
Gene-editing therapy for LGMD shows promise in mouse model
Jan 9, 2025 · Gene-editing therapy targets DYSF, whose mutations cause LGMD type 2B/R2. MD refers to a group of disorders characterized by progressively worsening muscle weakness and …
There’s never a dull moment living with Duchenne muscular …
Dec 18, 2024 · Living with Duchenne muscular dystrophy (DMD) has involved a series of challenges and triumphs, each shaping the person I am today. As 2025 approaches, I’m …
Educating all ages about living with muscular dystrophy
Oct 7, 2024 · In my four decades of life with limb-girdle muscular dystrophy, I’ve become comfortable educating everyone I meet about my quality of life and the ways all of us living …
New technology may inform effective MD gene therapies: Study
Nov 19, 2024 · Gene therapies for MD. Muscular dystrophy refers to a group of diseases that cause progressive muscle weakness and wasting. DMD, the most common type of muscular …
MDA honors researcher, patient advocate with 2025 Legacy Awards
Jan 30, 2025 · The 2025 MDA Legacy Award for Achievement in Clinical Research will be given to Katherine Mathews, MD, a researcher who has studied muscular dystrophy for decades, …