Daybue Stix (trofinetide), a powder formulation of Daybue (trofinetide) approved to treat adults and children ages 2 and older with Rett syndrome, may be used interchangeably with the original strawberry-flavored, ready-to-use liquid formulation. That’s according to results from a Phase 1 study showing that a single 12,000…
MIT researchers have discovered that tiny blood vessels in the brains of people with Rett syndrome may be more permeable, or “leaky,” according to a new study. Using miniature models of blood vessel systems built with cells derived from Rett patients, the team found that this leakage was driven…
Discussion
My last job at The New York Times, where I worked in various writing and editing positions for almost 30 years before retiring in 2023, was as an obituary writer. Almost all of the hundreds of obituaries I wrote were assigned to me by editors, who reviewed the daily list…
Discussion
My late daughter, Cammy, was diagnosed with Rett syndrome at just 20 months old in 2011. From that moment on, advocacy became part of our everyday life. Cammy’s journey taught our family not only about the challenges that people with disabilities face, but also about the importance of compassion,…
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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