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Pfizer Shares Update on Phase 3 Study of Investigational Mini-Dystrophin Gene Therapy
PPMD is deeply disappointed to share that Pfizer Inc. announced today that CIFFREO, a Phase […]
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While we’re all on unique paths, I welcome input from my community
Over the past couple months, I’ve shared my decision to try physical therapy in an […]
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Pitolisant found to reduce fatigue, daytime sleepiness in DM1 patients
After nearly three months of treatment with pitolisant, adults with myotonic dystrophy type 1 (DM1) […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
