All 25 participants in the Phase 3 Embolden study have received their doses of NGN-401, Neurogene’s experimental gene therapy for Rett syndrome, surpassing the trial’s initial enrollment target ahead of schedule. “We are pleased to have completed dosing in the Embolden trial of NGN-401 within our original timeline while…
Discussion
The Trenton Thunder’s baseball season opened the other day, though only a hardcore few noticed. Like most households in New Jersey, ours is fairly indifferent to the Thunder. And yet before the summer is over we will make several trips to see the team play, because watching a baseball game…
Discussion
In 2009, my husband and I were living in a third-floor condo in Chicago. My brother and his wife lived just 2 miles away. Within eight days of each other, we both welcomed beautiful baby girls into the world. It felt like a dream come true. Our daughters would grow…
Discussion
Stimulating the vagus nerve, part of the autonomic nervous system that regulates involuntary functions, paired with sound tones, may support the development of future therapies for communication deficits in Rett syndrome. Researchers found that VNS-tone pairing changed protein levels in the brain’s sound-processing region in a rat model of…
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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