We applaud a major legislative victory for the rare disease and Duchenne and Becker community — one made possible because advocates like you raised your voices, shared your stories, and sustained pressure on lawmakers.

With the signing of the FY 2026 Consolidated Appropriations Act (H.R. 7148) on February 3, 2026, Congress enacted a powerful package of funding and policy priorities with direct impact on our community. Here’s what that means for Duchenne and Becker families and rare disease advocates:

1. Rare Pediatric Disease PRV Program Reauthorized for Five Years

A top priority for the rare disease community — including PPMD — was ensuring the Rare Pediatric Disease Priority Review Voucher (PRV) Program didn’t lapse. This program has been a proven tool to spur investment in new therapies by granting a voucher to drug sponsors that can be used for expedited FDA review of another product.

What this means for Duchenne and Becker:

• The PRV program has historically helped advance therapies for rare conditions, including Duchenne and other neuromuscular diseases.
  
• Reauthorization for an additional five years provides predictability and incentive for companies developing treatments for children with Duchenne and other rare pediatric conditions.

This win was possible because advocates across rare diseases joined forces — making calls, sending emails, and telling legislators why this incentive matters. Your voice moved the needle.

 2. Federal Appropriations Include Duchenne and Rare Disease Funding

The FY 2026 appropriations bill also delivers critical federal investments that fuel research, innovation, and care for the Duchenne community. While not every disease receives a stand-alone line item, this package sustains robust support for the NIH, rare disease research programs, neuromuscular disease initiatives, and the broader health care infrastructure that advances lifesaving science.

Importantly, we secured $12.5 million for the Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP) and $8 million for the CDC’s Duchenne Muscular Dystrophy program. These targeted investments drive high-impact research, strengthen surveillance and data collection, and accelerate progress toward better treatments and improved outcomes for individuals living with Duchenne.

This year’s appropriations process was challenging, but your sustained advocacy ensured Duchenne and Becker and rare disease priorities remained front and center.

3. The Accelerating Kids’ Access to Care Act (AKACA) is Signed Into Law

Another landmark achievement in this legislative package was the inclusion of the Accelerating Kids’ Access to Care Act (AKACA) — H.R. 1509/S.752 — as a section within the appropriations bill.

This bipartisan law eliminates unnecessary red tape in Medicaid and CHIP provider enrollment:

• Out-of-state pediatric specialists who are already credentialed in another state can now enroll more easily to treat children covered by Medicaid/CHIP.
  
• This means faster access to specialized, time-sensitive care for kids with complex medical needs — like many in our Duchenne and Becker community.

Before AKACA became law, families often faced months of administrative delays when seeking treatment in another state — delays that can be life-altering for children in need of specialized interventions. Now, thanks to bipartisan support and your advocacy, we have a law that cuts through that red tape.

Why This Matters — And Why Your Voice Was the Difference

This legislative victory shows what’s possible when the Duchenne and Becker and rare disease community speaks with one voice:

• Policymakers responded to powerful personal stories and clear data about unmet needs.
  
• Bipartisan support emerged around practical, common-sense solutions.
  
• Long-term policy certainty now exists for the PRV program and improved access to specialized care.

Whether you called, emailed, met with your Member of Congress, shared your family’s story, or amplified messages on social media — you helped make this happen.

Looking Forward

While we celebrate this moment, our work is far from over. We will continue to:

• Monitor implementation of AKACA and ensure it delivers real results for families
  
• Advocate for sustained and expanded federal funding for Duchenne and Becker research
  
• Educate lawmakers about ongoing gaps in care, access, and innovation
  

And now, we build on this momentum.

Now is the time to sign up for PPMD’s 2026 Advocacy Conference this March. The progress we’ve achieved proves that advocacy works — and the next chapter starts with us showing up, together, in Washington, D.C. The relationships we build, the stories we share, and the priorities we advance at Advocacy Conference directly shape what becomes possible in the year ahead.

If this victory energized you, channel that energy forward. Join us. Bring your voice. Help us drive the next wave of progress for the Duchenne community. Register here.

Thank you for your tireless dedication — your advocacy is creating real, measurable change.