Dion Foundation urges Mass. delegation to support renewal of rare-disease legislation

To the Editor:

We reach out today as parents of three beautiful children, two of whom have recently been diagnosed with the ultra-rare neuromuscular disease limb-girdle muscular dystrophy 2C (LGMD2C). The sad reality is that these ultra-rare diseases affect a very small percentage of children, making development of treatments and cures incredibly expensive for pharmaceutical companies, without the promise of a commercial payoff at the end of the process.

We believe, as parents and co-founders of the Dion Foundation, that no child should be left behind. Our sole focus at the Dion Foundation is to increase awareness of rare genetic diseases that affect children, in order to fund research and development of potential treatments and cures of these devastating diseases. For years, Congress has supported rare pediatric disease research and development through legislation that awards priority review to drug companies that develop treatments for rare pediatric diseases. Without this legislation, there would be little to no incentive (beyond moral obligation) for these companies to focus on ultra-rare pediatric diseases like LGMD2C.

This crucial legislation is now up for its five-year renewal through the H.R. 7384 Creating Hope Reauthorization Act of 2024. We call on the members of the U.S. House of Representatives Energy and Commerce Committee to bring this measure to the floor, and we ask our Massachusetts U.S. representatives, as well as U.S. Sens. Ed Markey and Elizabeth Warren to advocate for this bill to ensure its passage. Our family’s hopes, and the hopes of many other children and their parents, rest on the renewal of this legislation.

Joseph and Courtney Dion

The Dion Foundation for Children with Rare Diseases

Marshfield

This article originally appeared on Telegram & Gazette: Dion Foundation urges MA delegation support rare disease legislation