All Blog Posts (1,678)

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…

On May 9, the Duchenne Regulatory Science Consortium (DRSC) held its Annual Meeting in Crystal City, VA. In the audience of about 40 people, there were industry and academic Consortium members, potential industry members, five FDA representatives, two NIH representatives, C-PATH representatives, and Buddy Cassidy, the DRSC…

The unknowns. Some of the hardest beasts to conquer in life.

The unpredictable variables that make life impossible to plan.

The not being able to commit to vacations and holiday plans because – well, you just don’t know what curve balls life will toss at you between…

This Mother’s Day, thank someone for their role in the fight to end Duchenne.

If you’re reading this blog, chances are you know my wife, Pat Furlong. For 23 years now, Pat has been leading Parent Project Muscular Dystrophy (PPMD) and the fight to end Duchenne. Many of you have told me over the years that Pat has been a source of comfort,…

PPMD is excited to unveil the agenda for our 2017 Connect Conference in Chicago, June 29 - July 2, 2017!

We are so proud of the …

Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. The community has anxiously awaited an update on both access and pricing ever since the deal between PTC and Marathon was announced.

On today’s call, we…

PTC Therapeutics has released a letter to the community and will host a conference call for the Duchenne community to discuss the launch plans for EMFLAZA™ (deflazacort) on Monday, May 8, 2017 at 3:00 PM EDT (call-in details are below).

On a…

The newest version of the GOP HealthCare Bill is coming to a vote by 1:30 PM EDT this afternoon!

This is moving fast and we need ALL MEMBERS of our community to …

PTC Therapeutics, Inc. will host a conference call for the Duchenne community to discuss the launch plans for Emflaza™ (deflazacort) on Monday, May 8, 2017 at…

Capricor Therapeutics today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne.

We are excited to see these results and to see the field of cell…

On May 18, 2017, FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s…