On Tuesday, March 8, 2022, Parent Project Muscular Dystrophy (PPMD) advocates met with scores of lawmakers to urge them to support programs important to the Duchenne and Becker community. PPMD is a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne),
For the second year running, PPMD’s 2022 Advocacy Conference was virtual, but no less impactful and important. It is estimated that more than 260 advocates participated in this year’s event, meeting with nearly 240 House and Senate offices to help inform Members of Congress about the critical need to continue investing federal resources in Duchenne-specific priorities and programs and to garner support on the BENEFIT Act.
This year’s PPMD Advocates Conference came shortly after it marked the 20th anniversary of the enactment of the Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Act in December, the law that ushered in two decades of increased resources and commitments to Duchenne research, public health, and therapy development efforts. PPMD’s Advocacy Conference is the longest-running advocacy event in rare disease in the United States, with over two decades of advocacy in Washington, DC.
“As we once again prepare to engage with Congress, the need for strong and sustained advocacy is no less than it was two decades ago,” said PPMD Founding President & CEO Pat Furlong prior to the event.
“While we are grateful for the progress that has occurred over the past 20 years, largely driven by the laws and policies PPMD advocates have helped drive, we know much more needs to be done. Our 2022 agenda is focused on ensuring federal programs keep pace with the needs in the field to lead to advancements in care, treatment, and overall life for families living with Duchenne,” she added.
PPMD Advocates Funding Request
This year’s Advocacy Conference included a request to continue funding the Centers for Disease Control and Prevention’s (CDC) Muscular Dystrophy program, which has focused on surveillance to better understand the prevalence of Duchenne, Becker, and other forms of muscular dystrophy, at $8 million and to fund the Department of Defense’s Duchenne research program at $12 million.
The request also sought to evaluate the impact care considerations and PPMD’s Certified Duchenne Care Centers have had on improving health outcomes and to identify remaining gaps and needs. And it urged support for medical research looking at the mental health challenges of patients and families impacted by Duchenne and to address challenges and opportunities in gene therapy for Duchenne.
In addition to these provisions, advocates also asked lawmakers to enact into law the bipartisan BENEFIT Act, S. 373 led by Senators Wicker and Klobuchar, and H.R. 4472 led by Representatives Matsui and Wenstrup, which aims to strengthen policies at the FDA focused on ensuring that the perspective of the patient and caregiver is considered as part of any review of candidate therapies.
PPMD has been a leader in the field of patient engagement over the past decade and believes the BENEFIT Act will further strengthen the field for Duchenne, Becker, and other communities.
“The Duchenne community has been tireless in its advocacy for over two decades, helping achieve enactment of multiple laws and other policies that have helped drive meaningful progress, including five FDA-approved therapies for Duchenne,” says Ryan Fischer, PPMD’s Chief Advocacy Officer
“As we head into our virtual fly-in, I thank our advocates for all you have done to get us to this point and all that you will do through your continued passionate advocacy. And I thank our many champions and supporters in Congress for moving us closer to our goal of ending Duchenne,” Fischer said prior to the PPMD advocates event.
[Source(s): Parent Project Muscular Dystrophy, PR Newswire]