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In 2001, Congress enacted the Muscular Dystrophy Community Assistance, Research, and Education (MD CARE) Act, transforming efforts to combat Duchenne muscular dystrophy. As a result, federal research commitments have expanded, leading to scientific breakthroughs and the development of potential therapies. These commitments have also leveraged significant non-federal investments, creating a strong public-private partnership. Additionally, the MD CARE Act has helped standardize patient care, significantly improving and extending the lives of individuals with Duchenne.

Continued progress depends on Congressional support. We ask for your leadership in advancing Duchenne research, patient care, and drug development efforts through the following actions:

Ask #1 - Sign the FY 2026 Appropriations Request for Duchenne

Thanks to Congress' leadership, significant progress has been made in the fight to end Duchenne. However, we must continue driving research and care programs at the federal level. This year, we request:

Centers for Disease Control and Prevention (CDC) – $10 million for muscular dystrophy programs to:
- Enhance muscular dystrophy research and tracking initiatives to improve Duchenne patient health outcomes (bone health, adrenal health, cardiac health, puberty, weight management).
- Examine successful newborn screening programs to inform national deployment.
National Institutes of Health (NIH) – Ensure data sharing to advance Duchenne research and increase transparency in the Muscular Dystrophy Coordinating Committee’s recommendations.
Food and Drug Administration (FDA) – Expand expertise in Duchenne to better understand therapy effects and address unmet needs through collaboration with patient and research organizations.
Department of Defense (DOD) – $15 million for the Duchenne-focused line item of the Congressionally Directed Medical Research Programs (CDMRP) to advance research and therapeutic development.

To support the FY26 Duchenne appropriations request, please contact:

Senate: Sen. Roger Wicker (R-MS)
House: Rep. Doris Matsui (D-CA) or Rep. Troy Balderson (R-OH)

Ask #2 - Protect Medicaid

Medicaid is a critical lifeline for individuals living with Duchenne and their families. Proposed cuts or restrictions would severely impact access to essential care, leading to increased hospitalizations and long-term costs. We urge you to:

Oppose Medicaid cuts, block grants, and restrictive work requirements that would limit access to specialists, treatments, and home care services.
Support policies that strengthen and expand Medicaid, ensuring that families affected by Duchenne continue receiving necessary medical and caregiver support.

Ask #3 - Reauthorize the Priority Review Voucher (PRV) Program

Duchenne is a rare, progressive, and fatal disease with no cure. The PRV program has been instrumental in incentivizing drug development for rare pediatric diseases by offering a valuable voucher to companies bringing new treatments to market. Since its inception, the PRV program has accelerated the development of life-changing therapies for rare disease communities, including Duchenne.

The expiration of the PRV program removes a critical incentive for companies to invest in rare pediatric disease research, delaying potential breakthroughs. We urge Congress to reauthorize the PRV program by passing the Give Kids a Chance Act of 2025 (H.R. 1262) in the House and a soon-to-be-introduced companion bill in the Senate.

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