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Support from Duchenne caregivers must include boundaries
Hawken Miller, who lives in Newport Beach, California, was diagnosed with Duchenne muscular dystrophy at age 5 in 2002. He shares why caregivers should support, rather than take over, when helping someone follow a treatment plan, and why building independence can help patients develop confidence in managing their own care. The post Support from Duchenne…
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Why judgment-free Duchenne noncompliance conversations matter
Edward Smith, MD, a neurologist, explains why he avoids judgmental approaches to noncompliance and instead focuses on trust, understanding barriers to care, and collaborative decision-making with patients and families. The post Why judgment-free Duchenne noncompliance conversations matter appeared first on Muscular Dystrophy News.
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Continued steroid use tied to slower lung decline in adults with DMD
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…
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What to Know Ahead of This Week’s Deramiocel FDA Advisory Committee Meeting
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 marks an important step in the FDA’s review process and provides an opportunity for independent experts to discuss the available…
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MDA Ambassador Guest Blog: The Power of Lasting Friendships
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…
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Simply Stated: Updates in Walker-Warburg Syndrome
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…
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Scientists discover a compound that could supercharge aging muscle repair
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.
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In our new home, we will have more space for our sons with DMD
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…
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Clinical Research Alert: Phase 3 Study of Salanersen in Older Teens and Adults with SMA
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…
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MDA Engage: Seminar connects neuromuscular community in Hershey
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…
