March 2024

A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular dystrophies.

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Among ambulatory boys with Duchenne muscular dystrophy (DMD), a one-time treatment with investigational gene therapy fordadistrogene movaparvovec has helped preserve functions and increase muscle volume for three years, especially in the youngest patients. That’s according to updated analyses from a Phase 1b trial (NCT03362502), in which the benefits of Pfizer‘s gene therapy candidate were most…

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This year’s Muscular Dystrophy Association Clinical and Scientific Conference was full of presentations on research, clinical trial updates, inspiring speeches, and networking among advocacy organizations, academics and industry representatives.   Gene therapy in Duchenne muscular dystrophy was a pervasive topic of discussion throughout the conference. Presentations focused on sharing experiences and learnings among clinicians who have…

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I don’t want to miss a thing. Yes, as I typed that, I sang the Aerosmith song in my head. It’s the soundtrack to my husband Jason’s favorite movie, “Armageddon.” But more than that, as a special needs parent and caregiver, I find that the lyric plays out repeatedly in my life. I have three…

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Long-term treatment with AOC 1001 continues to be safe and may reverse the progression of myotonic dystrophy type 1 (DM1), according to new data. A year of treatment eased myotonia, that is, the inability of muscle to relax after they’ve contracted, and improved muscle strength along with outcomes of activities of daily living, such as…

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RegenXBio shared additional interim data from their Phase 1/2 AAV micro-dystrophin gene therapy clinical trial for individuals with Duchenne aged 4-11 years old. The first participant in dose level 2, aged 12 years old, demonstrated 75.7% micro-dystrophin expression at 3 months. RegenXBio also shared video recordings of treated participants to indicate initial evidence of strength…

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REGENXBIO Inc. has shared an update on the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in patients with Duchenne ages 4 to 11 years old, including RGX-202 microdystrophin expression from dose level 2. RGX-202 is an an investigational gene therapy product for the potential treatment of Duchenne, utilizing a novel adeno-associated virus (AAV8) to transport…

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Over the past three decades, PPMD has driven Congress to enact legislation that has transformed federal public health, research, therapy development and access policies to bring us closer to ending Duchenne. This advocacy work has resulted in marked increases in Duchenne and related muscular dystrophy research funding, thorough public health surveillance to better understand the size…

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The PPMD Town Hall on Clinical Trials, taking place on Monday, March 4th during PPMD’s 2024 Advocacy Conference in Washington, D.C., aims to provide a platform for families affected by Duchenne and Becker to share their experiences and insights, with a focus on past, present, and future clinical trials. The primary goal is to hear…

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I’m reading “Eyes to the Wind: A Memoir of Love and Death, Hope and Resistance” by Ady Barkan, a late lawyer and political activist who had ALS. It’s been a tough read so far. In the book, Barkan recounts his experiences after being diagnosed with ALS in his early 30s. He does an excellent job…

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