Patient Advocacy Initiative staff speak on Notre Dame’s Rare Disease Day event and impact for the rare disease community.

Rare Disease Day is observed annually on the last day of February to raise awareness for the 300 million people worldwide living with a rare disease, their families, and carers. The day aims to improve the lives of those living with rare diseases by promoting equity in social opportunity,

Zevra used the awareness day to roll out the “Learn NPC, Read Between the Signs” disease awareness campaign. The biotech won FDA approval for a Niemann-Pick disease type C (NPC) treatment, Miplyffa, last year and recently agreed to sell a priority review voucher for $150 million to fund the launch and other work.

As we observe Rare Disease Day today, let us take a look at seven drugs poised to be approved to address rare diseases in 2025.

Every year since 2008, Rare Disease Day has been held on the last day of February to raise awareness about the research that goes into rare diseases like spinal muscular atrophy (SMA).

Rare Disease Day, observed on Feb. 28, brings attention to uncommon diseases affecting populations of 200,000 or fewer. In Roanoke, the Wells Fargo Tower will be illuminated in pink, green, purple, and blue—the colors of Rare Disease Day—thanks to a collaboration between The Fralin Biomedical Research Institute at VTC and Wells Fargo Tower.

Rare diseases affect multiple organ systems and approximately 50–75% of them affect children. Nearly 80% of all rare diseases are estimated to have an underlying genetic aetiology. The Indian Council of Medical