May 2024

Last Saturday, I attended a focus group on behalf of a local patient advocacy community of which I’m a member. The agenda for discussion was a nationwide allied health strategy here in Singapore to be implemented in coming years. For those unfamiliar with the term allied health, Kent State University’s College of Public Health refers…

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PPMD is excited to bring together experts in the cardiac field, spanning clinicians and researchers, to the third annual PPMD Cardiac Workshop, May 9-10, 2024 to delve into current and emerging issues in cardiac care in Duchenne and Becker muscular dystrophy. As part of PPMD’s Care Consensus Meeting Series, PPMD convenes leading experts to address…

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REGENXBIO provided a clinical and regulatory update on their Duchenne muscular dystrophy program highlighting progress in dose selection for their upcoming pivotal study which is expected to begin dosing patients in late Q3 2024 to early Q4 2024. In new data announced today from the second patient (aged 8.1 years) at dose level 2 from…

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Four-Day Conference Will Feature Key Opinion Leaders and Notable Speakers including Peter Marks, M.D., Ph.D., of U. S. Food & Drug Administration Newport Beach, Calif. (May 7, 2024) – CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, announced its FUTURES National Conference will be held from May 23-26 in…

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We are devastated to learn of the tragic loss of a young boy that was participating in Pfizer’s Phase 2 DAYLIGHT study. This study is evaluating the safety and dystrophin expression of fordadistrogene movaparvovec (PF-06939926), Pfizer’s gene therapy candidate for Duchenne, in boys who are two and three years old. According to the company, “We…

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PPMD, in collaboration with the Muscular Dystrophy Association (MDA), the EveryLife Foundation for Rare Diseases, and the Little Hercules Foundation (LHF), recently hosted a webinar on the background, history, and next steps for Duchenne Newborn Screening on both the state and federal levels.  What is Newborn Screening? Annie Kennedy, Chief of Policy, Advocacy, and Patient…

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A $250,000 award from the Parent Project Muscular Dystrophy (PPMD) will support the establishment of Baby Duchenne, a collaborative clinical research network for babies with Duchenne muscular dystrophy (DMD) diagnosed via newborn screening (NBS) programs in New York state. Under the direction of Bo Hoon Lee, MD, from the University of Rochester, the initiative seeks…

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Spring is by far the busiest season for my family. I have seven children who range in age from 2 to 23 years old, and three of them, Max, 18, Rowen, 15, and Charlie, 13, are living with Duchenne muscular dystrophy (DMD). Caregiving for my three sons with DMD takes a lot of my time…

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Disney Parks can be great places for someone with a power wheelchair to enjoy the various characters roaming around the park, rides that you can drive your chair onto, and parades and performances.  We recently visited Disneyland with two Disney fans in power wheelchairs to see how it can be done. While it takes extra…

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We are thrilled to welcome the newest addition to the PPMD team, Jodi Wolff, PhD, who joins us as our Chief Program Officer! Jodi brings with her a wealth of experience and an unwavering passion for advocating for individuals with rare diseases, particularly those with neuromuscular conditions such as Duchenne and Becker muscular dystrophy. Jodi’s…

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