PPMD is pleased to announce that late yesterday we submitted the results of our most recent patient-centered benefit-risk assessment (PCBR) study to the FDA. The assessment was done with research partners at the Johns Hopkins Bloomberg School of Public Health. PCBR assessment is an increasingly-favored…

Why Are Written Comments Important? How Are They Utilized?

Written comments are compiled and shared with the FDA Advisory Committee members prior to the date of a product’s/ company’s Advisory Committee meeting. Thus, written comments are a critical part of helping to provide context to the Advisory…

PPMD is proud to help author and share the attached letter to Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the FDA. Along with CDMD…

What’s a PRO?

Several patient-report outcomes (PROs) have been validated for use in people with Duchenne and their caregivers. Patient-report outcomes are a way for patients and families to provide structured input about their own experiences. PROs include a set of survey questions focused on…

This is our final update from the 2015 World Muscle Society meeting in Brighton, United Kingdom (click here for updates from previous days). Before providing summaries of some presentations on the last two days, we must share that this was an exciting meeting for…

Dr. John Porter and Kathi Kinnett from the PPMD team recently attended the World Muscle Society (WMS) meeting in Brighton,…

Dr. John Porter and Kathi Kinnett from the PPMD team are attending the World Muscle Society (WMS) meeting in…