Explore the latest insights, breakthroughs, and stories shaping our journey towards innovative research and advancements in STXBP1 and Single Gene disorders on Rafa’s Moonshot Blog.

When we embarked on this journey, we knew that we must think big for such a challenging mission. In the early days of Raphael’s diagnosis, Ella and I prepared our strategic plan and understood two important things: This led us to search for a scientific director to join and complete…
join the mission

Are you looking to make a meaningful impact in the lives of rare children? Your support will play a pivotal role in advancing science towards breakthroughs in rare genetic diseases, starting with STXBP1.

Are you pioneering a unique approach, developing novel methodologies, or advancing cutting-edge technology in the field of rare genetic diseases? Your expertise and innovation are crucial in our journey toward a breakthrough.