welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
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.
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 SkippingThis is a multicenter, open-label, dose-...
Catabasis Quarterly: Updates on edasalonexent and clinical trialsWe are pleased to announce that we have ...
Catabasis Pharmaceuticals Initiates Phase 3 PolarisDMD Clinical Trial For Edasalonexent In Duchenne Muscular Dystrop...Catabasis Pharmaceuticals, Inc., a clini...
Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System ...The aim of the proposed research is to c...
Jerry Mendell, M.D., Presented Positive Updated Results from the Four Children Dosed in the Gene Therapy Micro-dystr...Sarepta Therapeutics, Inc., a commercial...
DMD HUB: Expanding clinical trial capacity for Duchenne muscular dystrophy in the UKWith Duchenne muscular dystrophy (DMD) c...
MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dys...NF-κB is activated from infancy in DMD,...