Join us on Thursday, December 10, 2026, at 6 pm CT for an engaging CNS webinar focused on Making the Impossible Possible: A Natural History-Informed Framework for Single-Arm Studies of One-Time Gene Therapies in Rare Neurodevelopmental Disorders. This educational webinar will introduce Rett syndrome and the developmental plateau, then discuss the Plateau-Anchored, Baseline-Adjusted (PABA) framework for single-arm interventional studies, including studies of one-time gene therapies. The program will explore how natural history, participant-level developmental history, and baseline function can support rigorous and interpretable evaluation of treatment response. This is an industry-sponsored webinar presented by Taysha Gene Therapies, a CNS Mission Partner.
Speakers
Tessa Clarkson, PhD, Co-Founder and CEO of Psychlomere, LLC