Tuesday, January 31, 2023
Rett syndrome is a severe and progressive neurodevelopmental disorder that is characterized by a variety of neurologic and behavioral features. Although Rett syndrome is a rare disorder, it is the second most common cause of severe intellectual disability among females, with an incidence of 1 in 10,000 to 15,000. Because of the progressive nature of Rett syndrome, early diagnosis is critical for patients so they may receive appropriate interventions. Although treatment of Rett syndrome is currently limited to supportive care to address specific symptoms, newer interventions are currently in development.
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Why are investigational therapies targeting MECP2 or downstream pathways? Find out from Rett syndrome experts.
