With a Phase 3 trial currently in progress, Neurogene is getting ready to file an application seeking approval of NGN-401, the company’s experimental gene therapy for Rett syndrome. Neurogene recently completed dosing in the Phase 3 Embolden clinical trial (NCT05898620), which is testing NGN-401 in 25…
Discussion
Last in a series. Read parts one and two. When our daughter Abby, who has Rett syndrome, went into a Delaware hospital 17 years ago for spinal fusion surgery to correct severe scoliosis, she had an allergic reaction to something partway into the operation. The…
Discussion
Shape Therapeutics and the Rett Syndrome Research Trust (RSRT) are teaming up to advance the development of SHP-401, Shape’s experimental one-time gene therapy for certain people with Rett syndrome. RSRT will fund tests of SHP-401 in non-human primates to evaluate the therapy’s distribution in the body and…
Discussion
Second in a series. Read part one. Seventeen years ago right about now, my wife, Donna, and I sat dazed in a Delaware hospital as a surgeon explained that he had been forced to abort the spinal fusion he was performing on our daughter Abby because she’d gone into…
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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