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

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In honor of Rare Disease Day, the vice president of public policy and advocacy at the Muscular Dystrophy Association shared his perspective of advocacy and policy progress in the realm of rare neuromuscular diseases. [WATCH TIME: 6 minutes]

WATCH TIME: 6 minutes

"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, held on February 29, 2024, is a global event dedicated to raising awareness and understanding for the millions of patients who live with rare disorders. Although it may seem that patients with rare diseases are uncommon, research shows that their impact is collectively significant, affecting the lives of several families and communities worldwide.¹ This event serves as an important platform to amplify patient voices, advocate for increased research and support to one day enter into a future where rare diseases can be prevented, diagnosed earlier, and effectively treated.

In late January, Muscular Dystrophy Association (MDA)announced its Rare Disease Day campaign that aims to shed light on innovations in research, care and advocacy advanced by the organization to resolve the challenges faced by patients living with neuromuscular diseases such as muscular dystrophy, amyotrophic lateral sclerosis among others.² The organization’s work in advocacy for patients focuses on support for accelerated approvals, access to healthcare, newborn screening, accessible air travel and more.

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.

Registration for the 2024 MDA Conference is now open! The meeting is set to be held at the Hilton Orlando, in Orlando, Florida, from March 3 to 6, 2024. To register and for more information, head to www.mdaconference.org.

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REFERENCES
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. https://www.mda.org/press-releases/mda-launches-rare-disease-day-campaign