Paul Melmeyer, MPP, on Advocacy and Progress in Rare Neuromuscular Diseases From a Policy Standpoint


In observance of Rare Disease Day 2024, the vice president of public policy and advocacy at the Muscular Dystrophy Association discussed advocacy and policy progress regarding rare neuromuscular diseases.

This content originally appeared on our sister site, NeurologyLive®.

"This isn't just one day's effort or one week's effort or even just one month's effort. This is a constant effort that we, in the neuromuscular disease community, are trying to move forward."

Rare Disease Day is an annual event, held this year on February 29, focused on raising awareness and understanding for the millions of patients worldwide living with rare disorders. Despite the rarity of these diseases, research shows that their impact is collectively significant and affects the lives of many families and communities across the globe.1 Rare Disease Day serves as an critical platform to boost patient voices and push for more research and support with the goal of ultimately reaching a future where rare diseases can be prevented, diagnosed earlier, and treated effectively.

The Muscular Dystrophy Association (MDA)'s Rare Disease Day campaign, which was announced in January of this year, aims to bring attention to research innovations, care, and advocacy put forth by the organization with the intention of helping address the challenges faced by patients living with neuromuscular diseases, such as muscular dystrophy and amyotrophic lateral sclerosis.2 Support for accelerated approvals, access to healthcare, newborn screening, and accessible air travel are just some of the areas of advocacy the organization participates in.

Paul Melmeyer, MPP, the vice president of public policy and advocacy at MDA, recently sat down with NeurologyLive® to share his perspective on how Rare Disease Day helps to advance public policies for those with rare diseases. He also talked about significant initiatives expected during the White House Policy Forum on Rare Disease Day and the unveiling of proposed regulations for accessible air travel. Additionally, Melmeyer spoke about how MDA is working towards increasing access to care and therapeutic development for patients with neuromuscular disabilities.

Click here to register for the upcoming 2024 MDA Conference.

1. Cederroth H, Gahl WA, Landouré G, Zhang S, Bolz-Johnson M. Rare Disease Day: Amplifying voices, advocating hope. Med. 2024;5(2):103-105. doi:10.1016/j.medj.2024.01.003
2. Muscular Dystrophy Association Launches Rare Disease Day Campaign to Raise Awareness & Support for Neuromuscular Diseases throughout February. News Release. Muscular Dystrophy Association. Published January 31, 2024. Accessed February 27, 2024.
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