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EU panel recommends expanded use of DMD treatment Agamree
The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the European Union be expanded to include people with Duchenne muscular dystrophy (DMD) as young as 2. The CHMP is an arm of the European Medicines Agency (EMA) tasked with reviewing data on experimental therapies. The committee’s recommendation…
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Advancing Duchenne Newborn Screening: A Collaborative Path Forward
On April 24, 2026, PPMD, in collaboration with the Muscular Dystrophy Association (MDA) and the Little Hercules Foundation (LHF), convened a Duchenne Newborn Screening (NBS) Stakeholder Meeting in Philadelphia. The meeting’s main goal was to gather together industry partners in Duchenne to establish a foundation of understanding of the process of state implementation, and to…
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Expert Advice for Preparing for Medical Emergencies with NMD
Julianne Meiser, MSS, LCSW, an Outpatient Social Worker in the Division of Neurology at the Children’s Hospital of Philadelphia. By nature, medical emergencies are unexpected, stressful, and often traumatic experiences. For those living with neuromuscular disease (NMD), seeking emergent medical attention due to an injury or illness carries additional layers of important considerations. Depending on…
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Starting corticosteroids earlier helps improve motor function in DMD
Starting daily corticosteroids earlier in young children with Duchenne muscular dystrophy (DMD) helps them move better, walk or run faster, and keep their muscles stronger than delaying treatment by about one year, underscoring the importance of early diagnosis for preserving motor function. These are findings from a Phase 3 clinical study called FOR-DMD (NCT01603407), which…
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A tribute to a friend who taught us how to love, lead, and show up for others
Last Saturday, I lost a dear friend of mine, Timothy Chan, who had Duchenne muscular dystrophy. He passed away in the hospital in the morning, days after he had been admitted with a respiratory illness. The unexpected news from his parents shocked me, as I had caught up with him and a few friends from…
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Patient-led group launches network of clinics to improve LGMD care
Patient-led nonprofit The Speak Foundation has announced the launch of the LGMD Centers of Excellence, a novel alliance of clinics dedicated to improving care for people affected by limb-girdle muscular dystrophy (LGMD). “LGMD has historically faced significant barriers to therapeutic development due to fragmented care infrastructure and extremely small patient populations,” Kat Bryant Knudson, founder…
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Clinical Research Alert: Phase 3 Study of Omaveloxolone in Children and Teenagers with Friedreich Ataxia (FA)
Researchers at Biogen are seeking pediatric FA patients to participate in a phase 3 clinical trial to better understand Friedreich ataxia (FA) and to evaluate omaveloxolone, an investigational therapy for children and teenagers with FA. The study Children and teenagers with FA who are 2 to 15 years old may be eligible to participate in…
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Navigating disability benefits for muscular dystrophy
When the progressive muscle weakness of muscular dystrophy (MD) begins to interfere with your employment, the transition can feel overwhelming, but it doesn’t have to mean a loss of financial independence. Securing public disability benefits can provide the vital financial assistance and medical coverage needed to manage life with MD. By understanding how the condition…
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To me, a wheelchair is a symbol of improved function, not disability
Everyone recognizes the blue and white signs featuring a figure in a wheelchair that adorn certain parking spots. I call him the wheelchair guy, and he allows me to park close to public building entrances. The sign is an international symbol of disability. Of course, many disabled people are able to walk, but a wheelchair…
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Beyond Approval: Why Drug Development Doesn’t End at FDA Sign-Off
For many neuromuscular community members — and clinicians — who are waiting for a new treatment, a drug’s approval by the US Food and Drug Administration (FDA) marks the finish line. Years of discovery, testing, and clinical trials can result in a regulatory green light and, finally, patient access. But for researchers, FDA approval may…
