Rett syndrome is a devastating developmental disorder, principally occurring in girls, caused by mutations in the gene MECP2 that leads to severe cognitive, motor, and other symptoms. As such, the March 10 approval by the U.S. Food and Drug Administration of the first-ever treatment for the disorder, a drug called Trofinetide based on the natural protein IGF-1, brings new hope to patients and their families. The approval is also a dream come true for Mriganka Sur, Newton Professor of Neuroscience in The Picower Institute for Learning and Memory and the…
