News

On Sept. 6, I attended the launch of ReadSG at Singapore’s National Library to support my girlfriend, Amanda Yip. ReadSG is a five-year national movement by the National Library Board, Singapore’s public agency for libraries and archives, that encourages people to make reading an everyday habit, starting with just 15 minutes a day. It was The post Reading is one way I adapt to change with Duchenne MD appeared first on Muscular Dystrophy News.

In recognition of Muscular Dystrophy Awareness Month in September, the Muscular Dystrophy Awareness Month campaign features a series of stories highlighting the real-life experiences of people affected by muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, or X for more stories like this, using the hashtag #MDAwarenessMonth, or read the full The post Living fast and embracing everyday life with an ultra-rare type of MD appeared first on Muscular Dystrophy News.

This past weekend, PPMD headed west for PPMD Together: San Francisco, CA, bringing together families, clinicians, industry partners, and local organizations for two days of connection, learning, and collaboration. Following an evening reception on Friday,… The post Catch up on PPMD Together: San Francisco appeared first on Parent Project Muscular Dystrophy.

La Force DMD, Vaincre Duchenne Canada et Dystrophie musculaire Canada s’unissent pour un sondage national sur l’impact de la DMD. Vos réponses aideront à orienter les démarches auprès de la CDA-AMC et de l’INESSS concernant le givinostat. L’article Faites entendre votre voix. est apparu en premier sur La Force dmd.

An immune signaling pathway involving type I interferon (IFN-I) may be a potential therapeutic target for muscle damage in people with myotonic dystrophy type 1 (DM1), a new study suggests. An examination of immature muscle cells from children with DM1 found that increased IFN-I signaling was associated with stress in the cell’s protein-processing machinery and The post Immune pathway may contribute to muscle damage in myotonic dystrophy appeared first on Muscular Dystrophy News.

William Quickel lives in East Tennessee. He was diagnosed with a rare form of limb-girdle muscular dystrophy (LGMD) in 2021, when he was 27 years old. He has shared his story through his YouTube Channel and currently works for a local nonprofit that serves and supports teens and adults with autism. There was a time… The post MDA Ambassador Guest Blog: A Different Kind of Adventure – The Ups & Downs of Living with LGMD appeared first on Quest | Muscular Dystrophy Association.

I’m writing these words as we pass the 25th anniversary of the terrorist attacks of 9/11. Those of us old enough to clearly remember that day, and the days that followed, remember a nation and a world that was profoundly changed. Volumes have been written, studied, and analyzed in the quarter century since that terrible The post Disability inclusion and emergency-preparedness policy post-9/11 appeared first on Muscular Dystrophy News.

In recognition of Muscular Dystrophy Awareness Month in September, the Muscular Dystrophy Awareness Month campaign features a series of stories highlighting the real-life experiences of people affected by muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, or X for more stories like this, using the hashtag #MDAwarenessMonth, or read the full The post Nobody tells you the good part of life with muscular dystrophy appeared first on Muscular Dystrophy News.

In this episode of Quest Podcast, we chat with Sue Bruhn, PhD, CEO of the Charcot-Marie-Tooth Association (CMTA); Stephan Züchner, MD, PhD, Chief Genomics Officer at the University of Miami Miller School of Medicine; and Brian Lin, PhD, Senior Research Portfolio Director at the Muscular Dystrophy Association. Together, they break down what Charcot-Marie-Tooth disease (CMT)… The post Quest Podcast: More CMT Clinical Trials Than Ever Before: Inside the Research Turning Point appeared first on Quest | Muscular Dystrophy Association.

Newborn screening plays a pivotal role in early diagnosis and the opportunity to begin the right treatment and access clinical care within weeks of birth, sometimes before symptoms have even presented or begun to cause damage. With the ever-evolving neuromuscular disease treatment landscape and the availability of therapies that were non-existent twenty years ago, newborn… The post Newborn Screening & Early Access to Treatment: The Rogers Family SMA Story appeared first on Quest | Muscular Dystrophy Association.

Last week, I traveled with my sons Max, Rowen, and Charlie to Colorado for their multidisciplinary neuromuscular clinic visit at a children’s hospital. My husband and I typically don’t both travel with the boys, so one of us can stay home with our younger daughters. This time, it was my turn. I share seven children The post Transitioning my son from pediatric to adult DMD care is scary appeared first on Muscular Dystrophy News.

Earlier this week, PPMD joined global regulatory professionals, patient advocates, industry leaders, and others from across the healthcare community at RAPS Convergence 2026 in Charlotte, North Carolina. This year marked the 50th anniversary of the… The post PPMD Celebrates Patient-Centered Advocacy at RAPS Convergence 2026 appeared first on Parent Project Muscular Dystrophy.

Mindy Henderson has spent much of her life challenging the limits others have placed on her. When she was diagnosed with spinal muscular atrophy (SMA) — a genetic condition that causes muscles to weaken over time — as an infant, doctors told her parents that she might not live long and that school was not worth The post MDA Engage: Keynote speaker Mindy Henderson redefines the impossible appeared first on Muscular Dystrophy News.

A few days ago, my friend Reena Deen, a disabled independent filmmaker with complex post-traumatic stress disorder and dyslexia, shared with me that her film “The Damned Ones” was selected for the After Dark Program at the 2026 Torino Underground Cinefest. “The Damned Ones” is a mystery thriller about a blind university student named Siti The post A new film reminds us of the importance of disability representation appeared first on Muscular Dystrophy News.

Facioscapulohumeral muscular dystrophy (FSHD) and other health issues make it difficult for me to move these days. It’s hard to reposition myself when I’m sitting down. Standing to transfer now requires a seat that’s 22 inches or higher. It’s challenging to turn my feet to move from my wheelchair to the front seat of our The post FSHD is making it increasingly difficult to move my body appeared first on Muscular Dystrophy News.

In recognition of Muscular Dystrophy Awareness Month in September, the Muscular Dystrophy Awareness Month campaign features a series of stories highlighting the real-life experiences of people affected by muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, or X for more stories like this, using the hashtag #MDAwarenessMonth, or read the full The post Honoring the life and legacy of my brother, who had LGMD appeared first on Muscular Dystrophy News.

A newly discovered communication system in the microscopic “antennae” of cells may help explain how some congenital heart defects develop. When genetic mutations disrupt this system, the effects may extend beyond the heart to organs including the brain, kidneys, and skeleton.

As I prepared for college after graduating high school, my experience mirrored that of many young adults in several ways. Like my peers, I searched for a welcoming campus culture, a strong program in my intended major, and a university that offers meaningful opportunities for collaboration, research, and learning. However, my journey differed fundamentally because… The post Life with Lily: Preparing for College with a Neuromuscular Disease appeared first on Quest | Muscular Dystrophy Association.

The FDA has approved Isembyld (apitegromab-mstn), the first therapy designed to target muscle health and regeneration in people living with spinal muscular atrophy (SMA). This milestone adds to the progress made by genetic therapies for SMA and provides families with a new option focused on improving muscle strength and daily function. To help families understand… The post FDA Approves First Muscle-Targeted Therapy for SMA: A Conversation with Dr. Se-Jin Lee appeared first on Quest | Muscular Dystrophy Association.

Novartis announced today that the U.S. Food and Drug Administration (FDA) has notified the company that the Biologics License Application (BLA) of del-zota (delpacibart zotadirsen) is sufficiently complete to permit a substantive review. According to… The post Novartis Shares Del-Zota Regulatory Update: FDA Accepts BLA for Priority Review appeared first on Parent Project Muscular Dystrophy.

As an early funder of Avidity Biosciences, we are pleased to share that Novartis has announced that the FDA has accepted the Biologics License Application (BLA) for delpacibart zotadirsen (del-zota) for Priority The post FDA Grants Priority Review to Potential Duchenne Treatment Del-Zota 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.

Voting is more than a civic responsibility. It is a declaration of presence, power, and participation. The decisions made by elected officials touch nearly every aspect of our lives—from access to healthcare and medical research to community-based services, caregiver support, transportation, education, and employment. For people living with disabilities, however, participating in an election may… The post Your Voice, Your Vote appeared first on Quest | Muscular Dystrophy Association.

Kareem Azzazi is a 38-year-old content creator from Minnesota living with Duchenne Muscular Dystrophy. He uses eye-tracking technology to write, stream, play games, and create content online as TheSaturverse. Technology has always been more than a convenience for me. For some people, technology makes life faster or easier. For me, it often determines whether I… The post MDA Ambassador Guest Blog: Technology as a Tool for Independence appeared first on Quest | Muscular Dystrophy Association.

When looking at the list of accomplishments and accolades that musician and multi-platinum selling song-writer Donnie Demers achieved during his career, it is impossible not to be impressed by his talent and success – but what his family and loved ones remember most about the composer is his unwavering and genuine love for music and… The post Donnie Demers: The Man Behind the Music appeared first on Quest | Muscular Dystrophy Association.