The European Commission has approved Acadia Pharmaceuticals’ Daybu (trofinetide) — sold elsewhere as Daybue — as the first treatment authorized in the European Union for disease-related behavioral, emotional, and communication issues in people with Rett syndrome, ages 5 and older. The oral therapy, taken by mouth or via a…
Discussion
The area where I live in central New Jersey seems as if it’s becoming the warehouse capital of the country. For a decade or two, warehouses have been springing up like dandelions, some with recognizable names like Amazon on their signage, others offering no clue to passersby as to who…
Discussion
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…
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.
Get regular updates to your inbox.
