News

The U.S. Food and Drug Administration (FDA) is considering whether to conditionally approve zeleciment rostudirsen (z-rostudirsen), an experimental therapy for Duchenne muscular dystrophy (DMD) in patients with mutations that are amenable to exon 51 skipping. Z-rostudirsen developer Dyne Therapeutics announced that the FDA has agreed to review an application seeking accelerated approval of the investigational The post FDA set to decide on experimental DMD treatment in early 2027 appeared first on Muscular Dystrophy News.

Callie Hall lives in Orlando Florida and lives with a unique neuromuscular disability. She is passionate about disability rights, bluegrass mandolin, and being outdoors. RIIIIIIIING the school bell signals the end of the class period.  My teacher says, “Alright everyone, we have finished our week of learning about all the Civil Rights Movements.” Chairs shift… The post MDA Ambassador Guest Blog: What the Disability Movement Means to Me appeared first on Quest | Muscular Dystrophy Association.

Today, Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen) for the treatment of individuals living with Duchenne muscular dystrophy who… The post FDA Accepts Dyne Therapeutics’ Biologics License Application for Z-Rostudirsen appeared first on Parent Project Muscular Dystrophy.

CureDuchenne was an early funder of Dyne Therapeutics, and we are pleased to share that the FDA has accepted the BLA (Biologics License Application) for z-rostudirsen for individuals with Duchenne The post The FDA will review Dyne’s application for Accelerated Approval of z-rostudirsen for skipping exon 51 appeared first on CureDuchenne.

As the summer flies by, my thoughts turn to going back to school — reluctantly. As a mother of many, the end of summer is nothing new. I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie have Duchenne The post I wanted my oldest son with DMD to go to college more than he did appeared first on Muscular Dystrophy News.

Community members who use breathing devices say their ventilators support their freedom, mobility, and energy and improve respiratory health. The post Respiratory Support Makes a Big Difference: Community Perspectives on Using a Ventilator appeared first on Quest | Muscular Dystrophy Association.

Researchers have identified overactive HDACs as a key driver of muscle damage in DMD. By blocking these enzymes, HDAC inhibitors may help reduce inflammation, support muscle repair, and slow disease progression, making them a promising treatment approach. The post Understanding the role of HDACs in Duchenne progression appeared first on Muscular Dystrophy News.

Commendamide, a metabolite produced by beneficial gut bacteria that are depleted in people with Duchenne muscular dystrophy (DMD), protects muscle cells from damage by enhancing antioxidant mechanisms, a study found. “These findings provide new insights into the gut-muscle axis in DMD” and “support further investigation of microbiota-derived metabolites as postbiotic candidates for DMD therapy,” the The post Gut bacteria molecule guards against DMD muscle loss appeared first on Muscular Dystrophy News.

I’ve been forced to make lifestyle changes continuously because of the progression of my facioscapulohumeral muscular dystrophy (FSHD) and other health issues. But I’ve regained some independence in the past few weeks after losing some capabilities over the past six months. I have a new sit-to-stand cushion that helps me get up from my wheelchair more safely The post With FSHD, once one problem is solved, another soon takes its place appeared first on Muscular Dystrophy News.

Zach Fine and his family know firsthand how imperative it is to have an advocate to educate and fight for your needs while receiving medical care. Zach, a twenty-nine-year-old who lives with Duchenne muscular dystrophy (DMD), broke his femur two years ago and experienced severe complications during his emergency hospital stay. Throughout his hospitalization, his… The post How One Man with DMD is Using His Medical Emergency Experience to Educate Others appeared first on Quest | Muscular Dystrophy Association.

CureDuchenne, a U.S.-based nonprofit that funds research into new treatments for Duchenne muscular dystrophy (DMD), has launched a national public service announcement campaign to highlight the urgency behind research for people with DMD. The new advocacy campaign, dubbed A Cure Can’t Wait, highlights the need for aggressive work to find new treatments and, hopefully, a The post Advocacy campaign aiming for more birthdays for people with DMD appeared first on Muscular Dystrophy News.

With so many talented writers in our neuromuscular disease community, Quest Media is excited to share its third Spotlight on Community Authors. This series features a compilation of gifted and passionate authors sharing their stories, experiences, expertise, and imagination with readers of all ages. Whether you’re looking for historical fiction, mystery thrillers, Biblical studies, personal… The post Books to Add to Your Summer Reading List: Spotlight on Community Authors appeared first on Quest | Muscular Dystrophy Association.

Whether you joined us in Orlando or are catching up from home, the learning opportunities from PPMD’s 2026 Annual Conference continue. We’re excited to share that recordings and resources from PPMD’s 2026 Annual Conference are… The post Highlights from PPMD’s 2026 Annual Conference appeared first on Parent Project Muscular Dystrophy.

I wrote last week that I have been overwhelmed this summer by the demands of being a mom and caregiver. I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie live with Duchenne muscular dystrophy (DMD). Caring for The post I’m taking steps that will lead me away from caregiver burnout appeared first on Muscular Dystrophy News.

There’s a certain rhythm to gardening that I’ve always loved. The early mornings, the quiet work of turning soil, the satisfaction of seeing something grow because you cared for it. In my backyard, I’ve built raised beds filled with vegetables and herbs — nothing fancy, but enough to keep my hands busy and my mind The post I almost lost my garden to Becker MD. One tool brought it back. appeared first on Muscular Dystrophy News.

Two major steps are now complete in an early clinical trial testing a one-time epigenetic therapy — a treatment designed to turn on or off a specific gene without changing the underlying DNA — for facioscapulohumeral muscular dystrophy (FSHD), a genetic disease that characteristically affects the muscles of the face, shoulders, and upper arms. Epicrispr The post New milestones hit in MD trial testing treatment to turn off faulty gene appeared first on Muscular Dystrophy News.

Italfarmaco reported new data from the Phase 3 EPIDYS trial, showing that quantitative MRI measures of muscle mass and fat fraction were significantly different between the treatment and placebo groups.  The post Italfarmaco reports positive data from the EPIDYS trial and its ongoing open-label extension of givinostat in Duchenne appeared first on CureDuchenne.

Last month, the Purple Parade featured my story on its website. Reading my profile prompted me to reflect on why I continue speaking up about living with Duchenne muscular dystrophy (DMD) and what I hope these conversations might achieve. The Purple Parade is Singapore’s largest disability-inclusion movement, bringing people with disabilities, their families, businesses, community The post A Purple Parade profile amplifies my disability advocacy appeared first on Muscular Dystrophy News.

The post Satellos reports positive interim data from adults treated with SAT-3247 appeared first on CureDuchenne.

Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable… The post In Case You Missed It… appeared first on Quest | Muscular Dystrophy Association.

The FDA has scheduled an Advisory Committee meeting for July 29, 2026 to review Deramiocel, Capricor’s investigational cell therapy for Duchenne muscular dystrophy. The meeting will be held in person with the option to participate… The post Preparing for the Deramiocel FDA Advisory Committee Meeting: How to Submit Written Testimony appeared first on Parent Project Muscular Dystrophy.

Researchers at Teachers College, Columbia University (New York) are seeking children living with spinal muscular atrophy (SMA) who are receiving disease-modifying therapy to participate in a pilot study exploring the integration of targeted rehabilitation strategies. This intervention aims to strengthen muscles, enhance neuromuscular coordination, and reduce fatigue, ultimately supporting improved movement and functional abilities in children with… The post Clinical Research Alert: Clinical Study in Children with SMA appeared first on Quest | Muscular Dystrophy Association.

John Scurto is a 29-year-old MDA Ambassador from Boca Raton, Florida, who lives with spinal muscular atrophy (SMA) and is a power wheelchair user. He holds an MBA and a graduate certificate in Disability Studies, and he is especially interested in the intersection of business and disability inclusion. With both lived experience and academic training,… The post MDA Ambassador Guest Blog: The Part I Let Belong: Learning to Embrace My Disability Identity appeared first on Quest | Muscular Dystrophy Association.

More than 50 years ago, the Rehabilitation Act first outlawed disability discrimination and established a foundation for disability rights. The post MDA’s Guide to the Rehabilitation Act of 1973 appeared first on Quest | Muscular Dystrophy Association.

Proposed text:  CureDuchenne was an early funder of Sarepta Therapeutics, and we are pleased to share that the US FDA has accepted supplemental New Drug Applications (sNDAs) for AMONDYS 45 The post FDA accepts Sarepta’s application to convert AMONDYS 45 and VYONDYS 53 from accelerated to full approval appeared first on CureDuchenne.