Rafa’s Moonshot: Pioneering Cures for Rare Genetic Diseases
Rafa's Moonshot is a heartfelt mission to find a cure for STXBP1 disorders, rare genetic conditions that cause severe neurological challenges like developmental delays, epilepsy, and movement disorders. It began when Raphael, just 18 months old, was diagnosed with this condition. His parents, Sagi and Ella, launched Rafa's Moonshot to drive research and find solutions.
We work with top scientists to accelerate research and develop treatments, focusing on innovative approaches like repurposing existing drugs, gene therapies, and creating disease models. Our goal is to make progress both now and in the future—finding short-term treatments to help those affected today while paving the way for lasting cures.
Through our grant programs and collaborations, we're pushing the boundaries of current research. We believe that every step brings us closer to transforming the lives of Raphael and countless others affected by STXBP1. With hope and determination, we strive for a future where STXBP1 disorders can be effectively treated and, one day, cured.
Visit our website to learn more: https://rafasmoonshot.com/
Follow us on LinkedIn: https://www.linkedin.com/company/rafas-moonshot/