Cure Rare Disease™ is developing genetic medicines that are 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.The first-of-its-kind, CRISPR-based therapeutic aims to stop the progression of Duchenne muscular dystrophy in a single-patient dosing...
Cure Rare Disease™ is developing genetic medicines that are 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.The first-of-its-kind, CRISPR-based therapeutic aims to stop the progression of Duchenne muscular dystrophy in a single-patient dosing.
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