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…

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…

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…

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…

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…

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…

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.…

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…

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,…

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…

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.

They knew…

PTC Therapeutics has provided an update regarding EMFLAZA (see below). Please visit EMFLAZA.com for answers to some of the most frequently asked questions PTC has received.

Update from PTC…

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…