Celebrating Our 2022 Advocacy Accomplishments

From passing key legislation to our Virtual Summit and Hill Day, 2022 has been an exceptional year for MDA advocates. You raised your voices and key decisionmakers heard all of us loud and clear. Together, we achieved many legislative victories for the neuromuscular community.

As we look to 2023, let’s reflect on everything that we accomplished this year.

Access to Care

Thanks to our advocates, we vastly improved access to healthcare for many in the neuromuscular  community. Below are the key highlights.

• Multiple states added both Pompe disease and spinal muscular atrophy (SMA) to the list of conditions it screens all newborns for after birth. Now, at least 32 state screen for Pompe and 47 screen for SMA.

• MDA co-sponsored the nomination for Duchenne muscular dystrophy (DMD) to be added to the Recommended Uniform Screening Panel (RUSP) for newborn screening.
• Over multiple bills, Congress increased funding by $3 million for lifesaving newborn screening programs at both the Centers for Disease Control and Prevention and $2 million at the Health Resources and Services Administration. In addition, lawmakers funded $1 million for a study to improve and modernize the newborn screening program.
• Lawmakers extended key Affordable Care Act subsidies via the Inflation Reduction Act, which will make buying insurance more affordable.
• Starting in 2025, there will be a $2,000 per year out-of-pocket cost-sharing cap on Medicare prescription drugs.
• A two -year extension of policies that increased access to telehealth.
• Congress invested $450 million per year over three years in programs which will make it easier for people to access home and community-based healthcare services.

Accelerating Therapeutic Development

Together, we have made important strides to increase the speed of developing new therapeutics for the neuromuscular community. Below are our key accomplishments.

• Instead of the generic “Other Muscular Dystrophy” diagnostic code, those living with LGMD now have a diagnostic code that match their diagnosis. These codes potentially shorten the diagnosis timeline patients face, better delivers precise medical care, improves clinical trials, and increases future access to targeted treatments.
• Congress passed a suite of incredibly important new programs and initiatives for the FDA, including programs to create innovative new rare disease clinical trial endpoints, expedite regulatory review, and invest in gene and cell therapies. In addition, clinical trials will be more diverse, we saw improvements in the accelerated pathway for new therapies, and critical rare disease programs were renewed.
• Over multiple bills, legislators increased funding for the National Institutes of Health by $2.5 billion and $226 million for the Food and Drug Administration.
• We continue to successfully implement the ACT for ALS. Overall, this year MDA advocates secured $100 million in funding for the NIH expanded access grants program and $10 million in funding for the FDA rare neurodegenerative disease research grants program.

Empowerment & Independence

Over the last 12 months, MDA advocates repeatedly raised their voices in favor of policies that will truly empower the neuromuscular community. Check out these key accomplishments.

• Hundreds of advocates urged the Department of Transportation (DOT) to make lavatories more accessible on airplanes.
• DOT released an Airline Passengers with Disabilities Bill of Rights
• Advocates participated in first-person testimonials about their experiences traveling by air.
• Congress passed the ABLE Age Adjustment Act, which will increase the age by which an individual must have established a qualifying disability from 26 to 46.

Virtual Summit & Hill Day

Finally, after a three-year hiatus, advocates went back to Capitol Hill, but this time virtually. On September 28th and 29th, families from across the country raised their voices and urged lawmakers to take action on crucial issues impacting the neuromuscular community during the Virtual Summit & Hill Day. Together, we had 100 legislative meetings! Here is a quick recap of this empowering event!

Clockwise from upper right corner: MDA Staff Paul Melmeyer, Senator Susan Collins, Sen. Collins’ Staff Member Amanda Lincoln, and MDA Advocate Sarah Eslin.

We have accomplished a lot together this year and we couldn’t have done this without you! If you are not already an MDA grassroots advocate, join us today!