Cure Rare Disease is developing custom therapeutics that are as unique to the individuals they are meant to treat. Our mission is to offer effective, life-saving treatments developed through collaborations with world-renowned researchers and clinicians, and in partnership with our generous donors. Our customized therapeutics are designed specifically for the men and women who continue to fight for their right to live long, full, healthy lives despite having been diagnosed with a rare genetic disorder for which they’ve been told there is no treatment or cure.