A new study aims to raise awareness about Rett syndrome in boys and explore how the disorder affects them…
Articles by Marisa Wexler, MS
Treatment with Daybue (trofinetide) in clinical trials helped girls and young women with Rett syndrome to communicate and…
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.
Treatment with ADH-503, an experimental oral treatment for pancreatic cancer, was found to slow disease progression and prolong survival in…
Long-term treatment with Daybue (trofinetide) led to continual improvements among children and young adults with Rett syndrome in…
When clinicians have to decide whether to recommend invasive ventilation for adolescents with Rett syndrome, parental and familial support…
The U.S. Food and Drug Administration (FDA) has given a regenerative medicine advanced therapy (RMAT) designation to TSHA-102, an experimental…