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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…
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Our short film, ‘Lov-Able,’ heads to Australian disability-led film festival
On July 3, I received some deeply personal news: “Lov-Able,” a short film starring my girlfriend, Amanda, and me, was selected for inclusion in AN/OTHER Film Festival in Fremantle, Australia. This disability-led event is presented by DADAA, an Australian arts organization that promotes access to culture for persons with disabilities and mental illness. “Lov-Able” was…
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PPMD Honored with RAPS 2026 Patient-Centered Health Award
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 and regulation of medical products. For more than 30 years, PPMD has worked to ensure the voices of people living…
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MDA Ambassador Guest Blog: What the Disability Movement Means to Me
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…
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FDA set to decide on experimental DMD treatment in early 2027
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…
