"Kacper was diagnosed with surf1 deficiency in May 2018. Our whole life changed that day, but we knew we couldn't give up and we had to fight for medicine or therapy to save our son. Despite the illness that he has been through a lot, I do not give up, he is still a happy and smiling boy, and we strongly believe that our child and all children with Leigh Syndrome will receive their cure on time."
Rare Disease Cures Accelerator-Data and Analytics Platform
RDCA-DAP is an FDA-funded initiative that will provide a centralized and standardized infrastructure to support and accelerate rare disease characterization with the goal of accelerating therapy development.