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.
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
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