Q&A: The preclinical research origin of the first drug to treat Rett syndrome

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. That’s why the March 12 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.

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