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As I write this column, my wife, Wendy, is undergoing abdominal surgery. I won’t go into details, but it’s a pretty extensive procedure, so I’m concerned. I’d like to be there for Wendy, but due to my health issues, it’s just not possible. Instead, my daughter Jill is with her mom at the hospital. Wendy…

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Celebrate three decades of progress, community, and resilience at PPMD’s 30th Annual Conference, June 27–29, 2024 in Orlando, Florida! Back in 1994, our very first Conference was held in Orlando, and now, 30 years later, we are excited to return and commemorate the tremendous strides we’ve made together. We invite you to join us as…

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PPMD is pleased to learn of the U.S. commercial launch of AGAMREE® (vamorolone) oral suspension 40 mg/mL for the treatment of Duchenne in patients aged two years and older. According to Catalyst Pharmaceuticals, Inc., following the U.S. Food and Drug Administration (FDA) approval in October 2023, AGAMREE® is now available by prescription and dispensed throughout…

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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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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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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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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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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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