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 family optimistic for sisters with rare genetic disorder after FDA approves new drugs
AUSTIN, Oct. 6, 2024 (FOX & AUSTIN) – Two Austin sisters are fighting a rare genetic disorder, but the recent FDA approval of two new drugs is offering the family a promising path forward….Read More
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