NGN-401, a gene therapy candidate for Rett syndrome that’s now being tested in a Phase 1/2 clinical trial, was…
Articles by Marisa Wexler, MS
Treatment with NA-921 was found to improve measures of abnormal behavior and overall health in girls and young women with…
Dysfunction of an RNA molecule called NEAT1 contributes to nerve cell damage in Rett syndrome, a study suggested, implying…
A new study aims to raise awareness about Rett syndrome in boys and explore how the disorder affects them…
Treatment with Daybue (trofinetide) in clinical trials helped girls and young women with Rett syndrome to communicate and…
Mutations that cause Rett syndrome may disrupt the growth of brain cells during early development in the womb, according…
Acadia Pharmaceuticals has submitted a marketing authorization application for trofinetide to European Union regulators, seeking the therapy’s approval…
A new study sheds light on how MeCP2 protein dysfunction leads to brain abnormalities in Rett syndrome, and provides…
Health Canada has approved Daybue (trofinetide) for the treatment of Rett syndrome in adults and children ages 2…
Daybue (trofinetide) last year became the first treatment for Rett syndrome to win approval from the U.S.