Health Canada has approved a new treatment for Rett syndrome, DAYBUE, that has shown great promise in helping to limit symptoms and restore some parameters of health in children over the age of two living with the condition.
Rett syndrome is a debilitating disease affecting 600 to 900 Canadians. It starts in infancy and affects predominantly females.
Symptoms of the disease, which can include loss of mobility and hand function, slowed development and growth, loss of communication abilities, breathing problems, seizures, irritability, and unusual behaviours or moods, typically begin to show at 12 to 18 months of age.
The treatments available for Rett syndrome typically focus on addressing specific symptoms, such as anti-convulsants for seizures. Now,
The first and only treatment for Rett syndrome
DAYBUE, also known as Trofinetide, was created as a joint effort by Neuren Pharmaceuticals and Acadia Pharmaceuticals. It was approved by the U.S. Food and Drug Administration (FDA) in March of 2023 due to its clinical trial results showing great promise in helping to safely treat Rett syndrome symptoms.
The drug has been approved in Canada to be used in children over the age of two and adults living with Rett syndrome.
The positive results were based on the work done in the
. The trial, which included 187 participants between the ages of five and 20, showed that the drug passed safety checks and made significant changes in the presentation of symptoms of Rett syndrome.