Why It Matters
The Muscular Dystrophy Association (MDA) is pushing Congress to address barriers preventing patients with rare neuromuscular diseases from accessing affordable treatments and care. The MDA is lobbying for pharmacy benefit manager reform to reduce prescription drug costs, protecting Medicare and Medicaid coverage, securing robust research funding, and implementing newborn screening for early detection. These efforts represent a strategic bet that Congress will enact legislation addressing healthcare access, drug pricing, and research investment—areas where the MDA has identified concrete legislative vehicles and bipartisan support.
By the Numbers
The Muscular Dystrophy Association Inc. spent $250,000 on in-house lobbying in final quarter 2025, continuing a 17-year advocacy presence. The organization has filed 104 total disclosures worth $7.41 million since 2008.
MDA shifted to in-house advocacy in 2018 after using King & Spalding LLP from 2008-2017. In-house efforts have generated 68 disclosures totaling $6.37 million—significantly outpacing external counsel spending.
The current team includes Paul Melmeyer (25 disclosures worth $3.26 million since 2019, previously with NORD), Joel Shanley Cartner (14 disclosures worth $2.2 million since 2022), and Shannon Wood (6 disclosures totaling $580,000 since 2024).
The Agenda
The Muscular Dystrophy Association is pursuing several interconnected healthcare priorities in the fourth quarter of 2025.
The organization is pushing pharmacy benefit manager (PBM) reform for transparency and lower prescription costs, advocating for robust NIH funding while opposing caps on indirect research costs, and seeking to add Facioscapulohumeral muscular dystrophy to key research programs.
On patient access, MDA is working to expand Medicare and Medicaid coverage, extend Enhanced Premium Tax Credits, and increase access to home infusion services. The organization is championing the Newborn Screening Saves Lives Act following December 2025’s victory adding Duchenne Muscular Dystrophy to the screening panel.
MDA is also pursuing ALS Care Act reauthorization and improved transportation accessibility through the Air Carrier Access Amendments Act.
The advocacy aligns with active bipartisan efforts, including The CommonGround for Affordable Health Care Act with PBM transparency requirements and The Health Equity and Rare Disease Act of 2025 expanding NIH research.
Broader Context
MDA’s fourth quarter 2025 lobbying unfolds amid significant congressional momentum on healthcare issues affecting neuromuscular disease patients. Congress is actively debating PBM transparency with bipartisan support, while lawmakers engage in robust debates over NIH funding levels and Medicare access.
Recent legislative wins on newborn screening and emerging bipartisan interest in home infusion services create momentum for MDA’s priorities. Congressional champions like Senator Susan Collins and Representative Gus Bilirakis actively support rare disease initiatives.
However, the FDA’s restricted approval of Sarepta Therapeutics’ DMD gene therapy following safety concerns has intensified congressional scrutiny around accelerated approval pathways—a dynamic that could affect broader rare disease research policy.
Between The Lines
Congressional activity on MDA’s agenda is notably active across multiple fronts.
On PBM reform, both chambers have intensified scrutiny through committee hearings, with The CommonGround for Affordable Health Care Act including transparency requirements aligning with MDA priorities.
Medical research support appears strong. The Senate Appropriations Committee opposed NIH indirect cost caps, while Senator Collins secured $10 million for DMD research. The Health Equity and Rare Disease Act provides a vehicle for expanding rare disease research.
On Medicare and Medicaid access, Democrats are defending these programs, with Representative Matsui highlighting Medicaid as a lifeline for rare disease patients.
Competitive Landscape
MDA operates within a broader ecosystem of patient advocacy groups and pharmaceutical companies pushing overlapping priorities.
Sarepta Therapeutics Inc., specializing in DMD treatments, filed a $430,000 disclosure on overlapping issues including gene therapy innovation and patient access. PTC Therapeutics Inc. focuses on rare genetic disorders and regulatory pathways.
The National Organization for Rare Disorders (NORD) complements disease-specific advocacy. Notably, MDA’s Paul Melmeyer previously represented NORD from 2014-2019.
The Bottom Line
The MDA’s $250,000 final quarter 2025 lobbying effort targets PBM reform, NIH funding, Medicare access, and newborn screening expansion, capitalizing on bipartisan congressional momentum following December’s DMD screening panel addition. Led by veteran advocate Paul Melmeyer and operating alongside pharmaceutical allies, MDA is well-positioned to influence meaningful policy outcomes amid active healthcare reform discussions.
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