May 2017 Blog Posts (17)

FDA Makes Determination Regarding In-Dwelling Ports

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…


Added by Annie Kennedy on May 26, 2017 at 2:45pm — No Comments

Finding Your Light: Fighting Depression in Duchenne

PPMD's Kathi Kinnett, MSN, CNP worked with Molly Colvin, PhD, Neuropsychologist at Massachusetts General Hospital and Ginny Ward, an amazing mom living in Colorado with her children, including John, a young man living with Duchenne, to develop this important material. 

Life is hard, and parts of…


Added by Kathi Kinnett on May 25, 2017 at 11:00am — No Comments

Before Summer Kicks Off, Get Your Event Kicked Off!

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 an easy race to plan. Plus, the whole thing is over in less than 15…


Added by Nicole Herring on May 24, 2017 at 12:52pm — No Comments

FDA Pediatric Advisory Committee Unanimously Votes YES — Now Moves to Commissioner

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 clinical trial. This is an important step forward for our…


Added by Annie Kennedy on May 18, 2017 at 4:30pm — No Comments

Today’s FDA Pediatric Advisory Committee Meeting

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 arrive.

Because today – our…


Added by Annie Kennedy on May 18, 2017 at 10:00am — No Comments

PPMD Publishes & Testifies on Newborn Screening

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…


Added by Annie Kennedy on May 16, 2017 at 4:40pm — No Comments

PPMD Signs on to FDA User Fee Letter

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…


Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments

PPMD Helps Author Expanded Access Principles

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…

Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments

Senate Introduces BENEFIT Act

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…


Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments

2017 DRSC Annual Meeting

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…


Added by Abby Bronson on May 16, 2017 at 3:00pm — No Comments

Deciphering What We Know About EMFLAZA

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 now and then.


The not knowing whether you’ve…


Added by Pat Furlong on May 15, 2017 at 9:30am — 1 Comment

In Honor of Moms: A Note from Tom Furlong

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,…


Added by PPMD on May 10, 2017 at 10:30am — No Comments

PPMD's 2017 Connect Conference Agenda is Now Available!

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 …


Added by PPMD on May 9, 2017 at 11:00am — No Comments

PPMD's Response to PTC Announcement of EMFLAZA (deflazacort) Pricing & Access

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…


Added by PPMD on May 8, 2017 at 5:38pm — No Comments

PTC Therapeutics to Discuss Launch Plans for Emflaza™ (Deflazacort) during Community Call on Monday, May 8th at 3:00 PM EDT

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…


Added by PPMD on May 8, 2017 at 10:00am — 1 Comment

PPMD Response to the House Passage of the American Health Care Act

We may have lost the battle — but the war is far from over.

Some days are hard to make sense of. Today feels like one of those for many as we watched the House votes tally this afternoon.

This community knows better than most that our healthcare system needs repair and innovation. But the proposals that have been brought forward have not represented enhancements to healthcare…


Added by Annie Kennedy on May 4, 2017 at 6:00pm — No Comments

URGENT Action Alert – Call Congress Now Before This Afternoon’s AHCA Vote!

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 CALL your…


Added by Ryan Fischer on May 4, 2017 at 12:00pm — No Comments

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