PPMD is continuously seeking innovative opportunities to impact the process of developing therapies for Duchenne. Opportunity comes in many forms - funding promising research, advancing care standards for all patients, and advocating for key changes in federal policy.
Earlier this year Congress launched the 21st Century Cures Initiative, a comprehensive look at what can be done to accelerate the pace of cures in American therapeutic development. Congress is examining the entire pipeline, from discovery to development, to how therapies are ultimately delivered to patients. To do this, Congress has been engaging all stakeholders to identify where the existing gaps are in the pipeline, and then to seek input on how to close the gaps between advancements in scientific knowledge about potential cures and treatments, and the regulatory path of approvals for those cures and treatments.
The 21st Century Cures Initiative is in full swing and the Duchenne community has had a prominent seat at the table – literally. Over the past three months several members of the Duchenne community have participated in a series of Congressional Hearings and roundtables to inform members of Congress about the lessons learned in Duchenne and how improvements could and should be made to speed the development of potential treatments.
Below are some highlights from our involvement in 21st Century Cures over the past few months.
Congressional Hearing on Patient Perspectives
The Duchenne community has traveled a great distance over the past 15 years, thanks in significant part to the leadership of this Committee. But for too many families, my own included, this journey has not been fast enough, and we think every day about those whom we have lost.
We need treatments and therapies, right now, so we can end Duchenne and address the thousands of other rare diseases in need of treatments and cures.
Pat Furlong, PPMD Founder & President- Testifying to Congress at the 21st Century Cures hearing on Patient Perspectives.
Florida Regional Roundtable with Congressman Bilrakis
Each loss of function for our boys, including my son, is a "little death"... it is loss of function that he will not get back. The ripple affect from this is devastating. The negative impact on the family and the burden on the caregiver becomes increasingly significant and grows over time….
Working through the current regulatory issues and challenges that plaque our community, would be advantageous for all boys with Duchenne, including my brave little guy. Now is the time for change. Now is the time to help these boys and give them the bright future that they deserve!
Colleen Labbadia, parent of Brendan and PPMD FACES Coordinator - Speaking at the Congressman Bilrakis Florida regional roundtable
Illinois Regional Roundtable with Congressmen Shimkus & Davis
My family and I have watched Tim lose ambulation and now the ability to feed himself as he loses the use of his arms. Although a wheelchair can help with ambulation, there is no replacement for arm movement. Today boys like Tim with Duchenne are living longer than ever before, on average into their late 20s. But that is still far too short and simply unacceptable.
Our community is encouraged by the number of potential therapies that are in varying stages of development and evaluation, including three in critical late-stage clinical trials. We are hopeful that by the end of this year, the first-ever Duchenne drug applications will be submitted to the Food and Drug Administration, and we want the FDA to be ready to move as expeditiously as possible when this milestone occurs.
Ellen Wagner, parent of Tim and PPMD Board Member - Speaking at Congressmen Davis and Shimkus 21st Century Cures regional roundtable in Illinois.
We will continue to stay engaged with Congress and speak out about the need for a paradigm shift for rare diseases like Duchenne. The current path is broken for millions of patients living with rare diseases and we believe that the Duchenne example can also be applied as a template for other rare conditions that have not yet entered into late stage of drug development.
Our community continues to set precedents and we will not stop until we see life-saving treatments become a reality.