In April 2016, PPMD convened a workshop to assess the measures used to evaluate pulmonary strength, air flow and lung volumes. Thanks to the tireless effort of Dr. Jonathan Finder, this summary has now…Continue
Added by Kathi Kinnett on July 17, 2017 at 9:35am — No Comments
Sadly, I never had the honor of meeting PJ Nicholoff, but I’ve met his parents. And if PJ was anything like his father, Brian, he was vibrant, compassionate, caring, engaged and tenacious!
Added by Kathi Kinnett on July 16, 2017 at 8:30pm — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named Gillette Children's Specialty Healthcare Neuromuscular Clinic (Gillette) our 16th …Continue
Added by PPMD on July 13, 2017 at 11:21am — No Comments
Today, PPMD is convening medical experts from around the world for a workshop exploring inflammation and immunity in Duchenne on the eve of our 23rd Annual Connect Conference.
In January, PPMD organized a Critical…Continue
Added by PPMD on June 28, 2017 at 2:19pm — No Comments
An Overview of Your Impact in Action
We are less than a week away from PPMD’s Annual Connect Conference, where researchers and industry from around the world will provide…Continue
Added by Abby Bronson on June 23, 2017 at 2:00pm — No Comments
Navigating life with Duchenne can be daunting and time consuming for families. We rely on each other to share wisdom and information in order to make the process smoother and life a bit easier. The best guidance we can get is from those who have been through a given situation, sharing what they’ve learned to help others navigate resources that exist.
PPMD strives to find…Continue
Added by Ryan Fischer on June 21, 2017 at 10:00am — No Comments
Recently, FDA Commissioner Scott Gottlieb announced that the hiring freeze, instituted on January 23, has been lifted.
This reversal means that the FDA can begin to fill vacancies – needed resources for continued research into rare disease treatments and for the review and…Continue
Added by PPMD on June 15, 2017 at 11:53am — No Comments
With Congress not wrapping up spending bills for Fiscal Year 2017 until we were more than 7 months into the fiscal year, the FY 2018 budget and appropriations (funding) cycle will be interesting to say the least. The Trump Administration released a preliminary — or “skinny” budget -- in March but…Continue
Added by Ryan Fischer on June 15, 2017 at 11:30am — No Comments
Once again, PPMD has signed on to letters to leaders in both the House and Senate, requesting they continue to support the critical and highly successful defense health research programs funded through the Congressionally Directed Medical Research Programs (CDMRP) at the…Continue
Added by PPMD on June 15, 2017 at 11:30am — No Comments
This Father’s Day, I hope you will join me in celebrating the dads in this community. They are often the unsung heroes of our community. They are role models for our sons, providing for our families. Their hearts ache just as profoundly when our children are hurting. Their hearts love just as unconditionally.
That’s why this Father’s Day, I…Continue
Added by Pat Furlong on June 14, 2017 at 12:30pm — No Comments
PPMD's Pat Furlong
Earlier this month, we had the privilege of having our fourth stop in 2017 on …Continue
Added by PPMD on June 14, 2017 at 10:30am — No Comments
Last year advocates from Save Our Sons Duchenne Foundation (SOS) approached PPMD about partnering to have a patient report registry in Australia. They were worried because at the time there were no…
Added by Ann Lucas on June 5, 2017 at 9:30am — No Comments
Added by PPMD on June 2, 2017 at 11:30am — No Comments
Over the last 9 years, Coach To Cure MD has raised $1.5 million thanks to families like yours! Every dollar you have raised has helped us advance promising research, which led to two drug approvals for Duchenne in just the last year…Continue
Added by Danielle Garrigan on June 1, 2017 at 3:30pm — No Comments
Added by Kathi Kinnett on May 25, 2017 at 11:00am — No Comments
The Race to End Duchenne .1K is the perfect school event to celebrate Duchenne Action Month! About the length of a football field, a .1K is the race anyone can do! It requires no training and is…Continue
Added by Nicole Herring on May 24, 2017 at 12:52pm — No Comments
Today’s Pediatric Advisory Committee Meeting resulted in a unanimous decision from committee members to recommend the use of in-dwelling ports in Sarepta's ESSENCE…Continue
Added by Annie Kennedy on May 18, 2017 at 4:30pm — No Comments
As our clinical trial pipeline unfolds and therapies become available, navigating the landscape is becoming increasingly complex. But today our landscape shone a bit brighter as the sun rose on the FDA campus early this morning and we watched members of our Duchenne community…Continue
Added by Annie Kennedy on May 18, 2017 at 10:00am — No Comments
PPMD's SVP of Legislation & Public Policy, Annie Kennedy and Michelle Puryear, MD, PhD, testified at the Advisory Committee on Heritable Disorders in Newborns and Children last week.
On the heels of a recent publication in the International Journal of Neonatal Screening, Dr. Puryear provided an update of the therapeutic pipeline and some of PPMD's activities around developing infrastructure to support a pilot for newborn screening for…Continue
Added by Annie Kennedy on May 16, 2017 at 4:40pm — No Comments
PPMD is proud to join dozens of other rare disease nonprofit organizations in asking Congressional leaders to reauthorize the FDA user fee agreements.
The current FDA user fee agreements are the culmination of months of negotiation between FDA and the medical product industry, with significant input from the patient advocacy community.Continue
Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments