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Two young girls with Rett syndrome have been dosed with Neurogene‘s gene therapy candidate NGN-401 in a Phase 1/2 clinical trial, with data so far indicating that the treatment has been well tolerated. Another patient is expected to be dosed in the first months of 2024, with interim…
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I never imagined we’d be in the hospital for a second consecutive holiday, but we were. Our oldest daughter, Cammy, was admitted to the pediatric intensive care unit the day before Halloween. Now we’ve spent Thanksgiving in the hospital, too. But despite being here for a month, we…
Leriglitazone, an oral treatment candidate for neurological diseases, can improve the function of mitochondria — known as the powerhouse of the cell — in lab studies with cells from Rett syndrome patients and in a mouse model of the disease. Specifically, leriglitazone therapy restored energy production and reduced oxidative…
Effective communication is a top concern for people affected by Rett syndrome or related disorders, a U.S. caregiver survey has found. Other top concerns for patients include seizures, problems with walking or balancing, loss of function in the hands, and constipation. Recent progress in understanding brain development disorders such…
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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