Our goal is to develop novel tools and use them to discover fundamental mechanisms of brain wiring and processing, and how they go awry in disorders of brain development
On March 10 the FDA approved Trofinetide, a drug based on the protein IGF-1. The MIT professor's original research showing that IGF-1 could treat Rett was published in 2009.
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.