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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.
PhaseOut DMD: a Phase 2, proof of concept, clinical study of utrophin modulation with ezutromidThis study investigates the hypothesis t...
Strength Training in Duchenne Muscular DystrophyDuchenne muscular dystrophy (DMD) is a d...
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02This study, HALO-DMD-03, is a follow-on ...
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, ran...BACKGROUND:Duchenne muscular dystrophy i...
Clinical trial shows some promise for Duchenne muscular dystrophy drugThe results of an international clinical...
Sarepta Receives Negative CHMP Re-examination Opinion for EteplirsenSarepta Therapeutics, Inc., a commercial...
DMD HUB: Expanding clinical trial capacity for Duchenne muscular dystrophy in the UKWith Duchenne muscular dystrophy (DMD) c...