News

How the Fair Housing Act makes disability discrimination illegal in renting or buying a home and promotes access to permanent and temporary housing. The post MDA’s Guide to the Fair Housing Act appeared first on Quest | Muscular Dystrophy Association.

The University of Alabama at Birmingham (UAB) has launched an expanded access program to make the experimental exon-skipping therapy delpacibart zotadirsen (del-zota) available to qualifying patients with Duchenne muscular dystrophy (DMD). Del-zota, which is being developed by Avidity Biosciences, is specifically designed to treat DMD in people with disease-causing mutations that are amenable to exon The post Expanded access program to bring DMD treatment to patients in 1 US state appeared first on Muscular Dystrophy News.

In this episode of the Quest Podcast, we chat with Cerys Davage, a Welsh podcaster and content creator living with limb-girdle muscular dystrophy (LGMD), who has turned her diagnosis into a platform for connection and representation. Cerys opens up about growing up in a family that chose openness over silence, the moment in university when… The post Quest Podcast: Your Difference Is Your Superpower: A Conversation with Cerys Davage appeared first on Quest | Muscular Dystrophy Association.

An advisory committee to the U.S. Food and Drug Administration (FDA) has voted that available evidence does not support the effectiveness of the experimental cell therapy deramiocel for treating heart muscle disease in people with Duchenne muscular dystrophy (DMD). In a company press release, Capricor Therapeutics, the therapy’s developer, said the question before the committee The post FDA advisers reject evidence for Duchenne heart therapy appeared first on Muscular Dystrophy News.

Researchers at Carle Foundation Hospital in Urbana, IL are seeking individuals living with Becker or Duchenne muscular dystrophy (BMD/DMD) to participate in an observational study using Diffusion Tensor MRI to examine muscle function and structure. The study aims to better understand why muscles in BMD and DMD may be more prone to damage, and how… The post Clinical Research Alert: Observational Study of Muscle Function and Structure in Individuals with BMD/DMD appeared first on Quest | Muscular Dystrophy Association.

Chloe Crabb lives in Colorado with spinal muscular atrophy (SMA). She is a high school freshman and absolutely loves all things related to education and advocacy. Chloe lives with her parents, two cats named Mochi and Gracie, and her dog, Maggie. FUN FACT: Chloe loves sports cars and her dream car is a Porsche 918… The post MDA Ambassador Guest Blog: Life Beyond the Ramp appeared first on Quest | Muscular Dystrophy Association.

A protein once thought to mainly protect chromosome ends may also be essential for keeping muscle stem cells ready to repair injuries. Researchers found that TRF2 helps these cells maintain their identity and coordinate the cycle of rest, repair, and renewal.

Scientists have identified an immune response that may explain why statins cause muscle pain, weakness, and exercise intolerance in some people. The finding could eventually lead to treatments that protect muscles while preserving the drugs’ lifesaving cardiovascular benefits.

The first thing I tell others about myself is that my husband and I share seven children: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Being a mom is literally my favorite thing about myself. Caring for my family is my main purpose in life. Most mothers will The post A virtual FDA meeting gave me the opportunity to advocate for my sons appeared first on Muscular Dystrophy News.

The annual MDA Clinical & Scientific Conference is the largest global gathering of leading experts dedicated to the advancement of care and research for people living with neuromuscular disease (NMD). Each year, neurologists, researchers, industry partners, and patient advocates convene to share the latest scientific breakthroughs, current clinical trials, and evolving treatment landscape as they… The post Access, Impact, and Connection at the MDA Clinical & Scientific Conference appeared first on Quest | Muscular Dystrophy Association.

The next Clinical & Scientific Conference hosted by the Muscular Dystrophy Association (MDA) will take place March 21-24, 2027, in Orlando, Florida, and registration for the signature annual event — dubbed by the MDA “the leading international meeting dedicated to neuromuscular diseases” — is now open. The conference, to be held at Rosen Shingle Creek The post Discover new science in muscular dystrophy at the 2027 MDA Conference appeared first on Muscular Dystrophy News.

Today, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) met to discuss Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel as a potential treatment for Duchenne muscular dystrophy (Duchenne). Following a full day… The post Thank You for Raising Your Voices During the Deramiocel FDA Advisory Committee Meeting appeared first on Parent Project Muscular Dystrophy.

I returned home this past Saturday from my first time in a respite care facility, where I’d been staying for nine days. The backstory is that my wife, Wendy, and daughter, Jill, who are my primary caregivers, love to travel together. In the past, I’ve been able to manage daily living while they were away The post How respite care gives my family caregivers much-needed travel breaks appeared first on Muscular Dystrophy News.

On July 29th, 2026, the FDA will convene an Advisory Committee meeting to evaluate Deramiocel, Capricor Therapeutics’ investigational therapy for Duchenne muscular dystrophy. These meetings are an important step in the regulatory process, bringing together… The post Ahead of the July 29 Advisory Committee: We Urge the FDA to Consider the Totality of Evidence appeared first on Parent Project Muscular Dystrophy.

Continuing corticosteroid treatment into adulthood was associated with slower lung-function decline in people with Duchenne muscular dystrophy (DMD), according to a new study. “The results of this study provide further evidence to support continuing [corticosteroid] treatment into late adolescence and adulthood” for people with DMD, the researchers wrote. The study, “The Effect of Corticosteroids on The post Continued steroid use tied to slower lung decline in adults with DMD appeared first on Muscular Dystrophy News.

Edward Smith, MD, a neurologist, explains why he avoids judgmental approaches to noncompliance. The post Why judgment-free Duchenne noncompliance conversations matter appeared first on Muscular Dystrophy News.

Hawken Miller shares why caregivers should support, rather than take over, when helping someone follow a treatment plan. The post Support from Duchenne caregivers must include boundaries appeared first on Muscular Dystrophy News.

On Wednesday, July 29, 2026, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) will meet to review Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel as a potential treatment for Duchenne. The meeting… The post What to Know Ahead of This Week’s Deramiocel FDA Advisory Committee Meeting appeared first on Parent Project Muscular Dystrophy.

Cassidy Nilles is a 36-year-old living with LGMD 2J who lives in the suburbs of Chicago. Diagnosed at age 20, she started using a wheelchair after the birth of her daughter at 28. She is a single mom to her 8-year-old daughter Capri. Together they live in a multi-family home with her parents and her… The post MDA Ambassador Guest Blog: The Power of Lasting Friendships appeared first on Quest | Muscular Dystrophy Association.

In the last year, I have written about our family’s need for more space. Well, I have some exciting news to share. We are moving to a new house! It has been a process. We looked at houses for months. That might sound like we looked at a lot of houses, but we didn’t. In The post In our new home, we will have more space for our sons with DMD appeared first on Muscular Dystrophy News.

A sulfur-based compound called LASSS appears to protect and supercharge a key protein involved in repairing damaged muscle. The discovery could eventually lead to new ways to slow muscle loss and preserve strength as people age.

Walker-Warburg syndrome (WWS) is a rare, inherited disorder that significantly affects the muscles, brain, and eyes. It is a form of congenital muscular dystrophy (CMD), a group of genetic muscle diseases that become apparent within the first two years after birth. It is also the most severe form of a group of disorders known as… The post Simply Stated: Updates in Walker-Warburg Syndrome appeared first on Quest | Muscular Dystrophy Association.

Researchers at Biogen are conducting the phase 3 SOLAR study to learn more about spinal muscular atrophy (SMA) and to evaluate salanersen, an investigational therapy being studied for the treatment of SMA in individuals 15-60 years old. Salanersen is investigational and has not been approved by regulatory authorities. About the SOLAR study The SOLAR study is… The post Clinical Research Alert: Phase 3 Study of Salanersen in Older Teens and Adults with SMA appeared first on Quest | Muscular Dystrophy Association.

The Muscular Dystrophy Association (MDA) brought its Engage Community Seminar to Hershey, Pennsylvania, on July 18, uniting individuals with neuromuscular diseases, caregivers, and medical experts for a day of education and connection. Hosted in collaboration with the Penn State Health Milton S. Hershey Medical Center, the one-day event featured a variety of sessions to educate and The post MDA Engage: Seminar connects neuromuscular community in Hershey appeared first on Muscular Dystrophy News.

PPMD is honored to receive the 2026 Patient-Centered Health Award from the Regulatory Affairs Professionals Society (RAPS), recognizing organizations that have made significant contributions to advancing patient-centered healthcare and ensuring patient perspectives inform the development… The post PPMD Honored with RAPS 2026 Patient-Centered Health Award appeared first on Parent Project Muscular Dystrophy.