Parent Project Muscular Dystrophy (PPMD) has been invited to present at two upcoming stakeholder meetings.
The invitations, to PPMD Founding President & CEO Pat Furlong, were extended by leaders in the U.S. government in recognition of PPMD's groundbreaking work to advance the patient and parent perspective in the drug development and review…
Added by PPMD on September 10, 2013 at 9:44am — No Comments
Guest post by Jill Anne Castle, mom to Oliver, 11, and Anthony, 13 and living with Duchenne. Jill is an Educational Consultant and PPMD FACES of Arizona Volunteer Coordinator.…
The Muscular Dystrophy Coordinating Committee (MDCC) had its 10th meeting on August 26, 2013 at the National Institutes of Health offices in Bethesda, MD. The members of the MDCC come from federal agencies involved in one or more muscular dystrophy programs as well as public representatives from several organizations representing one or more forms of muscular dystrophy. There are representatives from…Continue
Added by Brian Denger on September 4, 2013 at 3:00pm — No Comments
It’s that time of year again – the start of another school year. While we put our faith in schools to provide a proper, inclusive education for our children, faith is not all we have in our arsenal for ensuring this is the case for students living with Duchenne.
As parents, you are…Continue
Added by Ryan Fischer on September 3, 2013 at 10:45am — No Comments
Saturday, August 17 marks the 15th Annual Mitchell's Run Thru Rockford. And for 15 years, the Peterson family has been raising money and awareness for Duchenne. Mitchell Peterson, 17 years old, has been the inspiration behind this event that continues to grow in numbers and momentum. He refuses to be defined by Duchenne and as he begins his senior year of high school, is already planning for a career after college. The Peterson…Continue
Added by Will Nolan on August 15, 2013 at 12:29pm — No Comments
Obtaining physical therapy (PT) services for individuals with Duchenne and Becker muscular dystrophy, and other conditions for which PT does not result in improvements in range of motion or function, has been a huge source of frustration for patients, parents, and providers.
Added by Kathi Kinnett on August 15, 2013 at 8:00am — No Comments
PPMD announced today that it will fund up to $1 million in projects by mid-2014 focused on the development of antisense oligonucleotides or other techniques to skip exons that are not currently in clinical development, or for multi-exon skipping projects, including the skipping of…Continue
“Hypertension” is the fancy word for high blood pressure. Blood pressure is the measure of how much pressure it takes the left ventricle of the heart to squeeze the blood out (the top number, or systolic pressure) and the pressure inside the left ventricle of the heart when the heart is relaxed (the…Continue
As we move into the start of 2014 and the February Advocacy Conference we are hopeful to gain momentum to move MD-CARE Act to committee action this year. With 78 cosponsors in the House and 17 in the Senate, we need your help to up our…Continue
Added by Ryan Fischer on July 31, 2013 at 9:30am — No Comments
Today is a landmark day for the entire Duchenne community. Not too long ago, the Duchenne therapy landscape was a barren field with little to no life. Thanks to a commitment to our patients and the field by the U.S. government, international governments, the venture community and industry, this landscape has changed markedly to the point where today we have the announcement for a…Continue
In 2008, PPMD joined the Genetic Alliance and others in the rare disease community, in support of “open access” – essentially ensuring that all NIH funded researchers make available to the public published peer-reviewed manuscripts and articles on a searchable website like PubMed Central or the National Library of Medicines. Making them freely accessible to the public within 12 months of publication.
Added by Ryan Fischer on July 18, 2013 at 4:30pm — No Comments
For six years now, the Dumm family in North Royalton, Ohio has been hosting JOA Picnic in the Park to raise money and awareness for Duchenne research. This is just one of the many things John Owen's Adventure, Inc. (JOA) does for our community. At the heart of JOA is 9-year-old John Owen Dumm who is a rising fourth grader with Duchenne and an active participant in his namesake event. In fact, John Owen and JOA have…Continue
Added by Will Nolan on July 16, 2013 at 3:03pm — No Comments
On July 9, PPMD will be meeting with individuals from FDA to include CDER, Division of Neurology, Office of Strategic Programs and Staff from the Office of health and Constituent Affairs.
The topics for discussion will include a review of PPMD’s Board Policy, developed in 2011. This policy embraces the following principles supporting an enhanced regulatory framework to advance development of drugs for rare diseases:
Added by Pat Furlong on July 3, 2013 at 1:30pm — No Comments
The Annual PPMD Connect Conferences have always been a haven for obtaining information around a broad range of topics applicable to the entire Duchenne community. Last year, we hosted a pre-conference meeting to address a single topic of need expressed by providers, parents, and patients alike – discrepancy in the care of people living with…Continue
Added by Kathi Kinnett on June 26, 2013 at 8:30am — No Comments
June 7/8, 2013
In February, 2013 EMA (Europeans Medicines Agency) released draft “Guidelines on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy” open for comment. This is the result of advances in basic and clinical research with an ever increasing number of clinical trials, recruiting small numbers of patients for these studies. This has raised a number of issues to include study design, the choice of appropriate…Continue
Coach To Cure, Sam's Day, North Texas FACES group, and now participating at PPMD’s Connect Conference at the end of June in Baltimore. These are just a few of the things 20-year-old Ben Dupree…Continue
Added by Will Nolan on June 14, 2013 at 12:39pm — No Comments
Earlier this week, PPMD hosted a webinar to discuss our FDA “aggressive collaboration” approach and the results from our recent benefit/risk survey. We conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments – information that PPMD is encouraging regulators to use to…Continue
Added by Holly Peay on June 14, 2013 at 10:00am — No Comments
With so many exciting happenings at PPMD and in our community, it is too easy to rush from one “fire” to the next and forget to celebrate our successes. Take a moment with us, and check out one success: our new Care for Duchenne pages.
We created the new…Continue