welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
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PPMD is committed to making research and development of new medicines for families living with Duchenne as fast and efficient as possible. Over the years, by listening to you, we have developed a clinical trials imperative that we strive to live up to.
Trials should not be limited to a subset of patients, but rather inclusive of young and old, ambulatory and non-ambulatory, those on steroids and those who are not.
Exposure to placebo should be limited or non-existent.
Trials should end quickly if they are not showing positive results and participants should be given another opportunity in a timely manner.
Efficiencies are needed across clinical trials, including contract negotiation, a single Institutional Review Board (IRB), standardized protocols and outcome measures, opportunities for combinations, and faster approvals.
As part of making these imperatives a reality, PPMD is evaluating a master protocol for Duchenne, along with community stakeholders, and has partnered with the Institute for Advanced Clinical Trials for Children (I-ACT) to address the complex issues that running a large trial with multiple therapies entails.
Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)This long-term extension study is an ope...
User-centred Assistive System for Arm Functions in Neuromuscular SubjectsRestore a lost function is a special exp...
Capricor trial failure in heart disease might offer another opportunityOn Friday Capricor Therapeutics announce...
Clay Matthews Supports Duchenne Muscular Dystrophy and First Approved Treatmenthttps://www.youtube.com/watch?v=WPu5pGEF...
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, ran...BACKGROUND:Duchenne muscular dystrophy i...
Edasalonexent could reduce functional decline in boys with DMD, MoveDMD phase 2 results suggestResults of the Phase 2 MoveDMD trial sho...
The Treatment of Duchenne Muscular Dystrophy: Equipoise, Fidelity and Standardshttps://www.youtube.com/watch?v=ji7Z23qH...