Rare Disease Isn't Rare
Over 30 million Americans are estimated to be living with a rare disease
Rare Disease Isn't Rare
There are over 7000 known ‘rare’ diseases
Rare Disease Isn't Rare
Receiving a rare disease diagnosis can be a multi-year ‘medical odyssey
Matthew McConaughey on the importance of the Firefly Fund
Matthew McConaughey shares a note on the Firefly Fund, a nonprofit organization based in Austin, TX. Our mission is to fund and support the research and education necessary to accelerate…Read More
Latest News & Events
Austin’s IntraBio gets FDA approval for Niemann-Pick Type C1 treatment
Oct. 01, 2024 (AUSTIN AMERICAN-STATESMAN) Six years ago, Austin sisters Belle and Abby Andrews were diagnosed with Niemann-Pick Type C1, a rare genetic disorder sometimes called childhood Alzheimer’s. Belle, who was 6…Read More
FDA approves Aqneursa for NPC
FOR IMMEDIATE RELEASE September 24, 2024 FDA approves Aqneursa for NPC Yesterday, the FDA approved AQNEURSA, a second therapy for NPC patients. This approval highlights the power of collaboration and…Read More