Added by PPMD on June 30, 2017 at 1:49pm — 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 Path Innovation Meeting with the FDA focused on…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
HOLA MI HIJO JOSE DAVID NECESITA DE SU APOYO SOMOS UNA FAMILIA DE ESCASOS RECURSOS ECONOMICOS DE CARTAGENA COLOMBIA ,ERAN DOS HERMANOS CON DUCHENNE YA FALLECIO UNO EL 1 DE DICIEMBRE DEL 2011 A LOS 17 AÑOS .POR LA FALTA DE UNA BUENA ALIMENTACION Y LA CASA EN MAL ESTADO LE DIO UNA FUERTE NEUMONIA EL CUAL LE PROBOCO EL PARO RESPIRATORIO...POR FAVOR AYUDEN A MI OTRO HIJO CON DUCHENNE JOSE DAVID VIAÑE DIAZ A QUE TENGA UNA MEJOR CALIDAD…Continue
Added by ramon ciriaco viaña ortiz on June 16, 2017 at 1:04pm — 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 approval of new medicines.
PPMD is proud to have been one…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 did not release a more complete budget until the end of…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 Department of Defense (DoD) for FY 2018.…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 PPMD's Every Single One Tour in Gainesville,…Continue
Added by PPMD on June 14, 2017 at 10:30am — No Comments
PTC Therapeutics, Inc. today announced that the FDA has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™). This is another important moment for our community and we look forward to the opportunity to share our collective experiences with Translarna with the FDA. As details on this Ad Comm become…Continue
Added by PPMD on June 6, 2017 at 8:47am — 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 clinical trials in Australia. This needed to change.
Added by Ann Lucas on June 5, 2017 at 9:30am — No Comments
Update from PTC…Continue
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