Parent Project Muscular Dystrophy Advocates Head to Capitol Hill to Advance Duchenne Public Policy Priorities

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Parent Project Muscular Dystrophy (PPMD)

Mar 06, 2023, 10:00 ET

2023 Aims Focus on FDA Reforms and Sustaining Surveillance, Medical Research and Patient Care Gains Achieved Over the Past Two Decades

WASHINGTON, March 6, 2023 /PRNewswire/ -- For the first time since 2020, advocates for Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will be back in person in Washington, D.C. for the organization's annual Advocacy Conference, March 5-7, to make their voices heard in the fight to end Duchenne.

Over 130 advocates – people with Duchenne, parents, siblings, grandparents, and other family members and friends will join PPMD on Capitol Hill for the annual meeting that continued on a virtual basis for the last two years during the COVID-19 pandemic. That persistence and commitment by advocates enabled PPMD and the broader Duchenne community to make continued progress on federal public policy priorities despite the challenging times and circumstances.

"There is no substitute for being face-to-face in Washington, D.C., especially this year. I am so grateful that once again our advocates will be able to gather in person for our annual Advocacy Conference," said Pat Furlong, PPMD Founding President and CEO.

"For more than 20 years, PPMD advocates have brought a unified voice to Washington, D.C. Through these advocacy efforts, our community began the journey that resulted in the enactment of the Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Act back in 2001, and together we have built upon and strengthened the federal commitment to Duchenne and broader muscular dystrophy research, public health, patient and caregiver support, and other activities. I am so thrilled we are back in person while also providing a virtual option for those who are unable to travel to Washington so they can still make their voices heard and foster relationships with members of Congress and staff," Furlong added.

This year's agenda will focus on bipartisan legislation to update current Food and Drug Administration (FDA) law to require that the all-important patient experience and related data points associated with candidate therapies be a core component of the FDA's benefit-risk assessment. This would build upon recent laws and enshrine experience data into the statute to provide a state of permanence while also providing additional transparency into how such information is being used by reviewers.

"Congress has done so much over the past two decades to create and refine the field of patient engagement and to ensure such inputs are used by FDA in a meaningful way. The bipartisan BENEFIT Act, sponsored by our longtime champions and friends Sens. Roger Wicker (R-MS) and Amy Klobuchar (D-MN) and Reps. Doris Matsui (D-CA) and Brad Wenstrup (R-OH), would achieve this goal. I urge Congress to enact this bill into law this year," Furlong said.

In addition to the BENEFIT Act, advocates will be urging their Representatives and Senators to support the annual Duchenne appropriations requests to strengthen medical research, public health, and patient care priorities. The 2023 (Fiscal Year 2024) request includes:

Increasing to $8 million from $7.5 million funding for the Centers for Disease Control and Prevention's (CDC) Muscular Dystrophy program to support muscular dystrophy surveillance and caregiver education, including ways to incorporate essential Duchenne care considerations into electronic health record (EHR) tools.
Reporting by the National Institutes of Health (NIH) on medical research programs focused on muscular dystrophy so the community can fully understand the work of the past two decades as well as increasing to $15 million the annual fund available through the Department of Defense to support Duchenne research.
Direction from Congress to advance consideration of adding newborn screening for Duchenne to the list of recommended tests to reduce the time to diagnosis and maximize the likelihood that all individuals with Duchenne can receive clinically recommended care as early as possible.

"For more than two decades, PPMD's Advocacy Conference has been an essential component of our overall advocacy strategy that has resulted in much progress for our community. While we have made many gains, we have much more to do and I am so thrilled that our advocates will be working so aggressively for continued progress," Furlong said.

About Parent Project Muscular Dystrophy

Duchenne is a genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won five FDA approvals.

Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.

SOURCE Parent Project Muscular Dystrophy (PPMD)