All Blog Posts (1,690)

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…

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…

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…

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 …

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…

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…

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…

Time and again in our Duchenne community, we see brave kids and their warrior parents work to change the landscape. After all, it’s that exact spirit upon which PPMD was founded. And with that same spirit that family after family has transformed their personal moments into broader movements that have benefited us all.

On May 18, the FDA Pediatric Advisory Committee convened to determine whether to allow for the protocol of Sarepta’s ESSENCE trial to…

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…

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…

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…

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…

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. 

Read the…

Earlier this month, PPMD was proud to be among seven patient advocacy organizations who took a lead in laying out a joint set of principles to guide any efforts that seek to change the process of accessing unapproved therapies outside of a clinical trial, also known as compassionate use or expanded access.

The patient advocacy organizations include:

• Alliance for Aging Research
• American Cancer Society Cancer Action Network
• Friends of…

PPMD is thrilled to announce that on May 4 the BENEFIT Act was introduced in the Senate by longtime Duchenne community champions, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN).

Over the past five years, Congress has made considerable progress in driving forward policies and procedures to ensure the patient perspective is considered by Food and Drug Administration (FDA) reviewers evaluating candidate drugs and other medical products. As a result of…