Building a Roadmap to Treat a Rare Neurodevelopmental Condition

Kleefstra syndrome is a rare neurodevelopmental condition with significant unmet medical needs, but a growing understanding of its underlying biology is creating new opportunities for therapeutic development. Eric Scheeff, chief scientific officer of Idefine and parent of a child with Kleefstra syndrome, discusses the organization’s progress in building the scientific foundation for a potential treatment, the role of its patient community, and the work ahead to translate scientific insights into meaningful therapies.