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Duvyzat recommended for EU approval as DMD treatment
The Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency known as CHMP for short, has recommended the approval of Duvyzat (givinostat) as a treatment for people with Duchenne muscular dystrophy (DMD), ages 6 and older, who are able to walk. The European Commission will review this recommendation, with a…
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Watch: Sarepta Therapeutics – Duchenne Gene Therapy Update (Webinar Recording)
Sarepta Therapeutics recently joined PPMD for a community webinar to share the latest updates on their gene therapy program. In addition to program updates, Dr. Chet Villa, cardiologist at Cincinnati Children’s Hospital, and Dr. Craig McDonald of UC Davis, an investigator in the EMBARK study of ELEVIDYS, offered clinical perspectives. Watch the Recording The post…
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The BELS Questionnaire: A New Tool to Support Your Child’s Emotional Wellbeing
PPMD is excited to share that a new tool, the BELS (Behavioral, Emotional, Learning, and Social) questionnaire, has been developed by Drs. Natalie Truba (Nationwide Children’s Hospital) and Molly Colvin (Massachusetts General Hospital) and was recently tested at Arkansas Children’s Hospital in partnership with Drs. Seth Sorensen and Aravindhan Veerapandiyan. While most doctors typically focus…
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Simply Stated: Updates in Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS) is a rare, progressive neurodegenerative disease that leads to muscle weakness, loss of physical function, and ultimately death, often within three to five years from symptom onset. ALS is caused by gradual degeneration of motor neurons, the nerve cells responsible for controlling voluntary muscles. As these neurons die, the muscles they control…
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MDA Ambassador Guest Blog: How the Power of a Few People (and a Dog) Can Change Your World
David Daw is 56 years old and lives in Kingston New York with his wife of 36 years. They have one daughter and four grandchildren. David was diagnosed with myofibrillar myopathy and spheroid myopathy. He is a musician, guitar tech, and dog lover. He and his wife currently have two Mastifrenchies ! I was officially diagnosed…
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Top 5 Things to Know About MDA’s Efforts to Protect Medicaid
In 2025, MDA’s advocacy team has focused on protecting Medicaid from Congressional budget cuts. We know many in the MDA community have questions about why Medicaid is important, current threats to the program, and how community members can get involved. We sat down with MDA’s Director of Advocacy Engagement, Mark Fisher, to learn the latest.…
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My son’s Make-A-Wish experience at WrestleMania was unforgettable
I’m writing this column as my husband, Jason, drives our family through the last rolling hills on the eastern side of the Rocky Mountains. Last week, we took an epic road trip from our quiet hometown in Nebraska to the eclectic sounds and neon lights of the Las Vegas Strip. Jason and I share seven…
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Why Multidisciplinary Care for ALS Yields Better Outcomes
For those living with neuromuscular diseases, including amyotrophic lateral sclerosis (ALS), access to quality care isn’t just important — it’s essential. The gold standard is the multidisciplinary care model, which is used at MDA Care Centers around the country. A multidisciplinary care approach leverages the combined expertise of specialists across disciplines to provide coordinated, patient-centered…
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CureDuchenne set to host 2025 Futures conference in San Antonio
CureDuchenne will host its annual Futures National Conference May 22-25 in San Antonio, as part of an effort to provide education and offer connections to members of the Duchenne and Becker muscular dystrophy communities. Under the theme “Together We Thrive,” the event will share information on research and resources for families affected by Duchenne muscular dystrophy.…
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Reflecting on 2 positive years of life, love, and Duchenne
Two days ago I marked the second anniversary of my relationship with my girlfriend, Amanda. As I celebrated this milestone, I found myself reflecting deeply on how much our lives have changed since we became a couple. Duchenne muscular dystrophy (DMD), my lifelong companion, has undoubtedly influenced our journey together. But instead of posing a…
