US biopharma company Acadia Pharmaceuticals has launched the LAVENDER phase 3 clinical trial to evaluate the efficacy and safety of trofinetide for the treatment of Rett syndrome in girls and young women.
Rett syndrome is considered to be a serious and rare neurodevelopmental congenital disorder related to the central nervous system (CNS). Symptoms of Rett syndrome are usually seen between six to 18 months of age, and result in problems with cognitive, motor, sensory, and autonomic function.
Jeffrey L. Neul – the investigator of the LAVENDER phase 3 clinical trial said: “There is no approved treatment for Rett syndrome, which is a rare neurological disease that impacts nearly every aspect of a child’s life, resulting in loss of speech, difficulty breathing, lack of motor control, loss of muscle tone and mobility, seizures, and more. Read more