Annie Kennedy's Blog (63)

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…

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…

Great news – The ABLE program enhancements have been re-introduced in Congress and are gaining support. This package of bills include improvements to the original ABLE program that directly impact our community, here’s how:

On April 4th, a bi-partisan group of Members of Congress,…

In late March, PPMD joined 87 organizations to issue a letter calling on Congress to take a hard look at the likely significant and…

PPMD is proud to sign on to letters sent both to the Senate and the House asking that they provide at least $29.8 million to the Centers for Disease Control and Prevention’s (CDC) Newborn Screening Quality Assurance Program (NSQAP) and at least $19.9 million to the Health Resources and Services…

Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can.

Priorities shared by all of us.

And to achieve this, PPMD has convened our industry partners through our Duchenne Drug Development Roundtable…

Later this spring – likely on or about May 18 – FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial.

Once again, our community is at a pivotal moment in our history where we…

Wow...what a week in Washington! President Trump released his budget proposal, the American Health Care Act moves to the floor of the House for a vote today, the U.S. Supreme Court ruled on a landmark case around the rights of students with disabilities within the public school system, and at long last – after months of our Duchenne…

“Who’s on first?”

Remember that classic Abbott & Costello routine? Well, trying to track the quickly-shifting healthcare bill has felt much like watching that routine. Complex, confusing, and exhausting. But the stakes here are no laughing matter.

We…

PPMD joined 200 patient organizations in a letter to President…

Years ago, a friend of mine named Tom Bailey who had Duchenne wrote a memoir that he entitled, Moving Mountains Without Muscles. As I look at the tremendous accomplishments of PPMD’s Adult Advisory Committee since we first launched this leadership group 2 years ago, I am astounded by how…

Prior to the approval of EXONDYS 51™, we knew that access to emerging therapies would be a process – and an often challenging process at…

The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts

To everyone in our PPMD community who called, reached out, emailed, and met with your elected…