CAPITOL HILL, WASHINGTON DC: Rare disease families gathered outside the U.S. Food and Drug Administration (FDA) in Washington, D.C. on Wednesday, March 18th, to hold a mock funeral, symbolizing the consequences of delays in access to treatments for progressive, life-threatening conditions.
The demonstration included a casket, hearse, and families carrying photos of children affected by rare diseases, including MPS1 (Hurler Syndrome). Advocates say that while scientific advancements are progressing, regulatory timelines are not keeping pace with the urgency of these conditions.
Families are calling on the FDA to use existing regulatory flexibility to accelerate access to safe and effective therapies for rare disease patients.
This footage was filmed by families participating in the demonstration.
Courtesy: Kennedy Ladd Foundation
https://kennedyladd.org
info@kennedyladd.org