Last week I participated in a briefing for Congressional staff in honor of the 10^th anniversary of the founding of the Critical Path Institute, our partner in the Duchenne Regulatory…

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 at…

The first annual meeting of PCORI was held in Washington, DC on October 6-8.  1,100 researchers, patients, caregivers, health system and industry representatives, and others were present to hear updates on patient-centered comparative clinical effectiveness research.  PCORnet is a “network of networks” that leverages databases and experience across diseases…

It’s fall, so it must be time for my annual mantra – Get Your Flu Vaccine!!

Everyone, 6 months of age and older, needs to be vaccinated against influenza, and protecting children with neuromuscular disease is especially important. A…

Stan Froehner’s group at the University of Washington recently reported preclinical efficacy data on the approved drug, Simvastatin, in the mdx mouse model of Duchenne (see PPMD's blog from the last day of the World Muscle…

After yesterday’s news regarding the Ad Comms for both BioMarin and Sarepta, the community continues to ask questions. PPMD has been working around the clock to get some answers and will continue to share information with you as it becomes…

Today we learned that the FDA will host an Advisory Committee (Ad Comm) meeting on November 24 to discuss the NDA for BioMarin’s drisapersen…

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 one…

Looks like we are just going to get into the Pfizer Myostatin Inhibitor trials with Hayden.  He turns 10 in just over a month and we're going for screening next week to Vancouver.  We were really starting to wonder if he was going to get in, but the folks at Vancouver pushed and unfortunately had to bump another boy that was scheduled to be the first at that location.  The other boy will still get in on the trial, but Hayden would have been excluded the day he turned 10 so they had to move…

Yesterday, I had the distinct privilege of representing PPMD and our Duchenne community as I sat before a full room of Senate staffers and congressional aides in the Dirksen Senate office building. Our goal was to urge them to press forward on the Innovation for Healthier Americans bill (also referred to as the ‘Innovation…

In the last few weeks we’ve heard the news from several companies working to develop therapies for Duchenne that they have received Rare Pediatric Disease Designations from the FDA. Santhera, Sarepta, Catabasis, and Biomarin.

We as a community have celebrated this news. But what does it actually mean?

And did you know that it…

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, UK…

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