FRAXA’s mission is to discover specific treatments and ultimately a cure for Fragile X syndrome, aiming to transition practical treatment into current medical practice quickly. They prioritize projects with clear, practical applications, emphasizing results sharing in a timely manner. By funding research grants and fellowships globally and partnering with biomedical and pharmaceutical companies, FRAXA bridges the gap between research discoveries and treatments, benefiting individuals affected by Fragile X and potentially those with autism, Alzheimer's, and other brain disorders.
FRAXA Research Foundation focuses on advancing research that promises to lead to new and improved treatments and ultimately a cure for Fragile X syndrome. They place particular importance on disease-modifying therapeutics derived from an understanding of Fragile X disease mechanisms. The foundation supports clinical trials as its highest priority, alongside translational research and early-stage investigations. FRAXA adapts its funding priorities based on the current research landscape and past bottlenecks in the research process, with a historical emphasis on overcoming issues like basic disease mechanism understanding and resource constraints. Recently, FRAXA has shifted its focus towards overcoming bottlenecks in preclinical validation and moving towards more efficient validation of promising drug treatment strategies to accelerate the pace of Fragile X research and treatment development.
Evaluation of applications will prioritize cost-effectiveness, giving an edge to proposals requesting fewer funds. Priority will be given to clinical trial proposals for agents that could alter the course of the disease and that have shown promise in Fragile X animal models, particularly those that have received prior support from FRAXA. While other clinical studies, including biomarker research in Fragile X patients or investigations into treatments for symptoms, will also be taken into consideration, they are regarded as secondary in importance.