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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


Firefly Fund: Shining a Light on Rare Disease

Firefly Fund is a global leader in providing crucial support for research and education necessary to accelerate cures for rare genetic diseases that affect children and currently have no cure.

Dan Rather speaks on behalf of the Firefly Fund

Your donations fund crucial support for research and education necessary to accelerate cures for rare genetic diseases that affect children and currently have no cure.

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


Our Lived Experience

Parents Pam and Chris Andrews noticed their eldest daughter Belle was showing developmental delays in gross and fine motor skills and coupled with other concerns they had such as a swollen spleen and low platelets, the parents and their young daughter began a multi-year diagnostic odyssey that spanned over a dozen therapists and medical specialists.

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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


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