S2E3 Monica Coenraads: The Fight to Cure Rett Syndrome

In this episode of Let’s Talk About That, Anirvan Ghosh speaks with Monica Coenraads, a transformative leader in the raredisease community whose work has reshaped the field of Rett syndrome, a severe neurodevelopmental disorder.Monica’s journey began as a parent, after her daughter Chelsea was diagnosed with Rett syndrome at a time when little was known about the disease and the causative gene, MECP2,had not yet been identified. Faced with limited answers and no treatments, Monica set out to understand the science—and soon recognized the need for a new, research-driven model.She went on to found the Rett Syndrome Research Trust (RSRT),raising more than $125 million to accelerate rigorous, collaborative science. A pivotal moment came in 2007, when research funded by RSRT demonstrated that restoring MECP2 in animal models could reverse Rett symptoms—even later in life—fundamentally changing how the disease was understood.In this conversation, Monica describes how RSRT built globalresearch consortia, catalyzed gene therapy programs now in the clinic, and is advancing next-generation approaches to cure Rett Syndrome. It is a powerful story about resilience, scientific focus, and how determined leadership can turn a devastating diagnosis into a credible path toward cures.