-
Gene therapy ATA-200 shows positive early results for children with LGMDR5
An experimental gene therapy called ATA-200 has shown promising safety and efficacy results in an early clinical trial for children with limb-girdle muscular dystrophy type R5 (LGMDR5). Long-term data from the first two treated patients suggest the therapy is working as expected, with no serious side effects, according to developer Atamyo Therapeutics. “These initial results…
-
Quest Podcast: The People Behind the People: Family Caregiving, Policy, and the Power of Showing Up
In this episode of the Quest Podcast, we chat with Nicole Lucas, a devoted family caregiver and dental hygienist who stepped away from her career so that her daughter could pursue her dreams; Carly Weber, a 24-year-old law student at the University of Pittsburgh living with spinal muscular atrophy (SMA) who has never let expectations…
-
MDA Ambassador Guest Blog: How to Find Community for Young People Living with Debilitating Diseases
Since her diagnosis with ALS at age 32, Gwen Petersen has poured her energy into advancing the science of her disease through participation in clinical and observational research. Gwen works together with her beloved group, Her ALS Story, to raise awareness that young people can get ALS, too. Gwen is married to her best friend,…
-
Monitoring Duchenne: Strategies for handling symptom changes
Living with Duchenne muscular dystrophy (DMD) means your loved one will need to adapt to symptom changes over time. Paying close attention to these changes is key because it can help your loved one maintain a sense of independence and control. It can also help you plan daily life adjustments and prepare for the future.…
-
Dyne initiates global Phase 3 trial in Duchenne amenable to skipping exon 51, ahead of planned FDA submission for accelerated approval in the US
Dyne Therapeutics, which received early funding from CureDuchenne, is initiating a Phase 3 FORZETTO Trial of z-rostudirsen in individuals with Duchenne amenable to skipping exon 51. Dyne is on track to submit a BLA to the FDA for accelerated approval in the US later this quarter, and the FORZETTO trial is intended to serve as…
-
In my life with DMD, I want to keep paying it forward, one story at a time
This week, I found myself reflecting on how much my life has changed since I joined Shalom Medcare, a care transport and medical escort provider in Singapore that was founded in 2021. Working there has become a part of how I advocate for healthcare equality for those who live with Duchenne muscular dystrophy (DMD), as…
-
CureDuchenne Announces Partnership with Tevard Biosciences to Advance Suppressor tRNA Therapy for the Treatment of Duchenne Muscular Dystrophy
Tevard Biosciences to present most recent data to Duchenne community for first time at CureDuchenne FUTURES National Conference on May 22 with webinar to follow on May 28 Newport Beach, Calif., May 20, 2026 —CureDuchenne, a global leader in funding and advancing research for Duchenne muscular dystrophy, today announced a second investment into Tevard Biosciences…
-
Dyne Announces Phase 3 Trial of Z-Rostudirsen, Plans for Submitting Biologics License Application to FDA
Dyne Therapeutics today announced the initiation of the company’s Phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, DYNE-251), in individuals living with Duchenne who are amenable to exon 51 skipping. Z-rostudiresen is an investigational exon skipping therapy bound to an antigen-binding fragment (Fab) to help the therapy reach the muscle cells for those amenable to…
-
CureDuchenne Appoints Lidia Gardner, PhD, EMBA, as Vice President of Clinical and Medical Affairs to Advance Patient-Centered Care, Research, and Strategic Partnerships
NEWPORT BEACH, Calif., May 20, 2026 /PRNewswire/ — CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, proudly announces the appointment of Lidia Gardner, PhD, EMBA, as its new Vice President of Clinical and Medical Affairs. Dr. Gardner brings more than 15 years of experience spanning clinical strategy, patient advocacy, evidence generation,…
-
RGX-202 gene therapy meets main goal in Phase 3 DMD clinical trial
The Phase 3 portion of a clinical trial testing RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD), met its main goal, the therapy’s developer, Regenxbio, announced in a press release. Steve Pakola, MD, Regenxbio’s chief medical officer, said the data “support the potential of RGX-202 to become a best-in-class gene therapy for Duchenne…
