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Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy
As a community, we are crushed to hear the news from Pfizer that their Phase 3 mini-dystrophin gene therapy trial in Duchenne failed to meet its primary endpoint. Individuals, aged 4 to 7 years old, treated with the experimental gene therapy did not show significant improvement in motor function, as measured by the North Star…
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Consensus Statement Featuring Expert Insights from PPMD’s Certified Duchenne Care Centers on Access to Novel Therapies for Duchenne Published
PPMD is excited to share that a consensus statement providing insights from clinicians who care for patients with Duchenne into the current therapeutic landscape and access to novel therapies for Duchenne has been published in the Annals of Child Neurology Society. The landscape of approved therapies in Duchenne is rapidly changing with eight approved therapies,…
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Roche Announces Phase II Open-Label SHIELD DMD Study to Assess Effect of Satralizumab on Bone Health in Duchenne
Today Roche announced plans for the SHIELD DMD study, a phase II open-label study to investigate the use of satralizumab in ambulatory and non-ambulatory individuals with Duchenne. Satralizumab is a monoclonal antibody that inhibits interleukin-6 and is an approved therapy for adults with neuromyelitis optica spectrum disorder. According to Roche, with the understanding of the…
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During a busy season, we must take things one day at a time
I feel relaxed — refreshed, even. We’ve had almost two weeks at home without any appointments or travel. The kids are complaining of boredom, but I’m letting boredom reign! It’s a beautiful but rare occurrence for my family of nine. My husband, Jason, and I have seven children: Lexi, 23, Max, 18, Chance, 17, Rowen,…
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Summer Fun: Accessible Activities for All Ages and Interests
As the school year wraps up, we are all looking forward to a fun-filled summer. It can be overwhelming to plan outings and activities considering accessibility and necessary accommodations to keep everyone safe. Along with your ideas and feedback, PPMD has compiled a list of ideas for all ages and interests: Spend time…
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Advisory board named to shepherd Duchenne MD candidate
Satellos Bioscience has established a clinical advisory board, with experts in drug development and genetic muscle disorders, to help propel SAT-3247, an oral therapy candidate for Duchenne muscular dystrophy (DMD). “The formation of this clinical advisory board marks a major development step for Satellos as we continue our evolution in becoming a clinical stage drug…
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Three years of CAP-1002 still stabilizing arm, heart function
After three years of treatment with Capricor Therapeutics’ experimental cell therapy CAP-1002, people with Duchenne muscular dystrophy (DMD) continue to show benefits in arm and heart function, new data from the HOPE-2 open-label extension (OLE) study shows. Topline data from the Phase 3 HOPE-3 trial (NCT05126758), which is testing the therapy in DMD patients, ages…
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Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002
Capricor Therapeutics has announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study of CAP-1002 for the treatment of Duchenne. CAP-1002 is the company’s novel cell therapy. The HOPE-2 is a double-blind, randomized, placebo-controlled trial in ambulatory or non-ambulatory patients with Duchenne. It is the first trial to use the Performance of Upper…
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Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy
Read the full news release HERE Capricor Therapeutics, which received early funding from CureDuchenne, has announced positive 3-year results from the HOPE-2 open-label extension study. Individuals receiving CAP-1002 continue to show upper-limb and cardiac benefits after 3 years of treatment, compared to an external control group of similar DMD patients. Results from the pivotal Phase…
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What’s it like to have three sons living with DMD?
What’s it like to have three children with Duchenne muscular dystrophy (DMD)? That’s the question I’m asked more than any other, as a mom and caregiver to three sons with DMD: Max, 18, Rowen, 15, and Charlie, 13. Of course, that question is only asked by parents or grandparents of children with Duchenne. I assume…
