“DiMe’s focus on Core Measures for Pediatric Rare Disease is invaluable for CURE SYNGAP1 and so many other patient advocacy groups who are heading into Clinical Trials for their gene-based medicines. We will be using this carefully curated resource to find effective endpoints and to help us show the FDA what is possible to measure based on our deepest concerns for our children’s health. This is the missing link for all the clinicians, advocates, and drug developers, who currently are looking for a cure but need each other’s expertise.”

– Kathryn Helde, PhD | Chief Scientific Officer, CURE SYNGAP1