PPMD Community

Annie Kennedy's Blog (72)

2017 World Muscle Society: Highlighting Progress in Duchenne

“WMS - What’s the buzz?”


It has been many years since I’ve had the opportunity to attend the World Muscle Society meeting and wow how things have changed! While PPMD has strong representation here every year, this year I have…


Added by Annie Kennedy on October 4, 2017 at 1:00pm — No Comments

ACTION ALERT: Stop the Graham-Cassidy proposal in the Senate

The Senate is considering another effort to repeal the Affordable Care Act (ACA) before the end of the month. Last week, Senators Lindsey Graham (R-SC), Bill Cassidy (R-LA), Dean Heller (R-NV), and Ron Johnson (R-WI) released a new plan to repeal and replace the ACA. This proposal is commonly referred to as…


Added by Annie Kennedy on September 19, 2017 at 10:00am — No Comments

PPMD Lends Leadership to NCATS Rare Disease Research Toolkit Development

There are some days when I feel like our community is in its infancy. When I look at where we are in terms of access, therapeutic options, combinations, care implementation…

But more often than not, I am overwhelmed with gratitude for the infrastructure and resources…


Added by Annie Kennedy on September 13, 2017 at 5:00pm — No Comments

MD Community Moves Forward ICD Code Nomination for Duchenne/Becker

Paperwork – oh the paperwork!

Each time you visit a physician’s office or hospital, we leave with piles of forms and papers detailing the visit. The providers seen, the prescriptions written, results from tests conducted, instructions for follow up, and more. And each of those pieces of…


Added by Annie Kennedy on September 13, 2017 at 10:00am — No Comments

PDUFA VI Passes the Senate – Now Moves to President’s Desk to be Signed Into Law

The PDUFA VI bill – known as FDA Reauthorization Act in the House (FDARA) – passed the House of Representatives early in July with strong bi-partisan support. It then moved to the Senate but consideration was delayed until after the Senate healthcare reform deliberations were completed. Today, though, we are excited that a companion bill (mirroring the House version) passed in the Senate! It will now move to the President’s desk where it is expected…


Added by Annie Kennedy on August 3, 2017 at 8:30pm — No Comments

The BENEFIT Act Passes the Senate!

Today – the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, S. 1052 – led by Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) passed in the Senate!


This means that – thanks to the leadership of our Senate champions and our Duchenne…


Added by Annie Kennedy on August 3, 2017 at 12:33pm — No Comments

UPDATE - Action Alert: Your Calls Are Still Needed! Tell Your Senator to Vote NO on the Senate Health Bill

Yesterday the Senate voted 51-50 to move forward on healthcare reform legislation -- but no specific bill was actually voted on. The Senate has committed to passing something this week. Once passed, the Senate bill will then have to return to the House for passage or conferencing (if it differs from…


Added by Annie Kennedy on July 26, 2017 at 9:00am — No Comments

ACTION ALERT: Tell Your Senator to Vote NO on Today's Healthcare Repeal!

Today, the Senate is expected to start floor debate and motion for a vote on either the Better Care Reconciliation Act (BCRA), which would repeal and immediately replace the Affordable Care Act (ACA), or the Obamacare Repeal Reconciliation Act (ORRA), which would repeal portions of the ACA after a two-year delay. The Senate is working to pass whichever bill is more likely to attain 50 votes. 


The Congressional Budget Office released reports last week that…


Added by Annie Kennedy on July 25, 2017 at 9:05am — No Comments

FY 18 Labor HHS Report Language Released: Our Duchenne Community is Stronger Than Ever!

Transitions are hard. That’s the truth whether you’re talking about a new school year, a new job, or even a new Congress. And, in particular, new Presidential Administrations are always complex political climates in Washington.


But this year, our Duchenne community came together once again and embraced the challenge of this latest transition. With so many new Members of Congress and Federal Agency leadership in flux, we knew that our 2017 February…


Added by Annie Kennedy on July 19, 2017 at 12:00pm — No Comments

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

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…


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…


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

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

ACTION ALERT: ­Immediate Action Needed – Revised AHCA Bill Threatens Duchenne Community’s Access to Health Care Coverage

The current version of American Health Care Act (AHCA) directly threatens essential health benefits (EHBs), Medicaid eligibility and funding, and vital health care protections for our Duchenne community. 


I wish there was a way to…


Added by Annie Kennedy on April 28, 2017 at 9:30am — No Comments

Members of Congress Re-Introduce ABLE Improvement Bills

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,…


Added by Annie Kennedy on April 14, 2017 at 9:00pm — No Comments

PPMD Joins Letter to Protect Medicaid

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


Added by Annie Kennedy on April 13, 2017 at 9:30pm — No Comments

© 2017   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service