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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.
Design of a phase 3 trial to evaluate the long-term efficacy and safety of ataluren in patients with nonsense mutati...Ataluren is conditionally approved by th...
Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patien...This study is done to evaluate whether t...
A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)The purpose of the study is to see wheth...
Catabasis Pharmaceuticals Announces Publication Of Phase 1 Clinical Results Of Edasalonexent (CAT-1004) In Duchenne ...Catabasis Pharmaceuticals, Inc., a clini...
Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study ReportsVamorolone, a Duchenne muscular dystroph...
Catabasis Quarterly: Updates on edasalonexent and clinical trialsWe are pleased to announce that we have ...
Roche Letter to the Duchenne CommunityBy now, many have heard about Pfizer's d...