Many types of movement disorders such as uncontrolled muscle contractions and Parkinson’s-like motor changes often co-occur with Rett syndrome, a study shows. The severity of movement disorders is associated with Rett’s overall severity, with uncontrolled muscle contractions particularly linked with breathing trouble and digestive problems, data indicate. The findings…
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Neurogene has received written agreement from the U.S. Food and Drug Administration (FDA) for a registrational trial of NGN-401, a gene therapy candidate for Rett syndrome. A registrational, or pivotal, trial is designed to provide the evidence needed to support an application for FDA approval. “We appreciate the…
Having a child with Rett syndrome who can’t communicate their needs, desires, and emotions as easily as a neurotypical person does is sad, frustrating, and complex. There were many times when we asked our late daughter, Cammy, a series of yes-or-no questions to figure out what was going on…
An aspiration event— when something other than air enters the lungs — increases the risk of lower respiratory tract infection and respiratory failure in people with Rett syndrome, a study found. Patients with known or suspected aspiration events more commonly had risk factors like epilepsy and difficulty swallowing, and…
Rett syndrome is a rare genetic disorder that mostly affects females. The disease is characterized by normal early development, followed by slowed growth and a loss of motor control. Learn more about the disease here.
Most cases are caused by a mutation in the MECP2 gene. Scientists have identified mutations in CDKL5 and FOXG1 genes in those who have atypical or congenital Rett. Learn more about what causes the disease here.
Rett syndrome is associated with problems in cognitive, sensory, emotional, and motor skills, and across the nervous system that controls involuntary (or autonomic) functions. Learn more about the symptoms of the disease here.
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