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In total, 42 have been published by Muscular Dystrophy News which Ground News has aggregated in the past 3 months.

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Top Muscular Dystrophy News News

FDA Meeting Gives Window Into Gene Therapy Field’s Angst

FDA · United States

FDA Meeting Gives Window Into Gene Therapy Field’s Angst

100% Center coverage: 2 sources

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.See the Story

European Commission approves treatment for Duchenne muscular dystrophy

Brussels · Brussels

European Commission approves treatment for Duchenne muscular dystrophy

Italfarmaco has announced that the European Commission (EC) has granted conditional marketing authorisation for Duvyzat (givinostat), a novel histone deacetylase (HDAC) inhibitor.See the Story

After incredible hardships, I still feel blessed

Shanksville · Shanksville

After incredible hardships, I still feel blessed

I’m between two anniversaries. Today is the 36th anniversary of the collision I had with a drunk driver that cost me my eyesight and almost cost me my life. Saturday will be my 45th wedding anniversary. The car crash happened on the evening of June 11, 1989. I was returning home from playing music at a 50th wedding anniversary celebration. My wife, Wendy, and I had planned to leave early the next morning for our annual vacation with her sister a…Read Article

Viral vector in LGMD2E gene therapy gets new FDA designation

The FDA granted platform technology designation to the viral vector used in the LGMD2E gene therapy SRP-9003.United States · United States Read Article

We’re our own best experts, but what if we’re incapacitated?

I believe that many adults living with rare diseases can and do become experts in their conditions, sometimes more so than healthcare professionals. With respect to my own journey with limb-girdle muscular dystrophy, this point became particularly clear when I had a near-death experience in early 2024. Several types of muscular dystrophy (MD) are rare, including my own form of it. This makes it difficult for me to find healthcare professionals w…Boston, Massachusetts · Boston Read Article

Fear and pride as my son starts up with high school band

When I think about my son Charlie, I always remember my pregnancy with him. I’d taken a home test and knew I was pregnant, but when I called to make an appointment with my doctor to confirm the good news, I scheduled an appointment for my son Max, who was 4, at the same time. Our family doctor had received a note from Max’s physical therapist and wanted to discuss it with us. The physical therapist’s note led to a diagnosis of Duchenne muscular …Mississippi · Mississippi Read Article

Pepgen discontinues development of Duchenne MD exon-skipping therapy

Pepgen will stop development of its Duchenne MD exon-skipping therapy PGN-ED051 after increases in dystrophin levels proved disappointing.Mississippi · Mississippi Read Article

Why those of us with FSHD feel like we’re always in transition

During a recent facioscapulohumeral muscular dystrophy (FSHD) wellness group meeting via Zoom, we each shared how we were feeling about our disease journey. One participant said, “I feel like I’m always in transition.” I can relate to that. With FSHD, the need to adapt seems never-ending, a topic that has frequented my thoughts over the last few months. I thought about jokingly asking if folks were interested in joining a new organization called…Read Article

A day of reflection leads me to a place of gratitude as a DMD mom

Once again, I’m writing from an uncomfortable chair in an infusion room as my son Max participates in a clinical trial. I frequently visit this room with my three sons — Max, 19; Rowen, 16; and Charlie, 14 — who live with Duchenne muscular dystrophy (DMD). It’s a boring day. Well, let me take that back. Parts of the day are long and tedious because we’re sitting in this small square room, probably no more than 12 feet by 12 feet. I find myself l…Omaha, Nebraska · Omaha Read Article

Hurdles Remain Despite Progress in Gene Therapy for DMD

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further innovations in safety, efficacy, and treatment strategies are needed.See the Story

DMD treatment SAT-3247 safe in trial, shows early signs of efficacy

Satellos Bioscience’s SAT-3247 was safe, well-tolerated, and showed initial signs of increasing muscle strength in five adults with Duchenne muscular dystrophy (DMD), the company said. “We believe the findings from this Phase 1b study support our plan to advance SAT-3247 into a placebo-controlled Phase 2 trial,” Satellos CEO Frank Gleeson said in a company press release. The company said it plans regulatory submissions in the third quarter for a…Australia · Australia Read Article

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