This website provides information and support for families affected by Rett syndrome. You will find here information about Rett New Zealand, a brief description of Rett syndrome, links to current research and links to other useful sites including links to disability support services. We have also added a page for personal stories and other contributions for families to share their experiences.
Latest News
July 17 2023
Licensing agreement for trofinetide expanded
Acadia Pharmaceuticals Inc. (NASDAQ: ACAD) today announced that it has expanded its current licensing agreement for trofinetide with Neuren Pharmaceuticals to acquire ex-North American rights to the drug as well as global rights in Rett syndrome and Fragile X syndrome to Neuren's development candidate NNZ-2591.
Breakthrough neurological drug comes from Dame Margaret Brimble's lab
Distinguished Professor of Chemistry, Dame Margaret Brimble. Photo: Chris Loefte
A molecule discovered by Distinguished Professor Dame Margaret Brimble of the University of Auckland is the basis for a breakthrough neurological drug approved by the US Food and Drug Administration on March 10.
Trofinetide will treat Rett syndrome, a condition causing symptoms similar to cerebral palsy or autism in an estimated one in 10,000 girls.
More than 20 years after the molecule was discovered, Trofinetide, to be marketed as Daybue, is the first drug treatment for the condition.
FDA Approves Trofinetide for Treatment of Rett Syndrome!
Friday 10 March 2023 was a milestone day for our community, more than a decade in the making. Acadia Pharmaceuticals announced that Trofinetide, marketed under the new name DAYBUE™, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. This is the first treatment ever approved for individuals with Rett, a drug that has demonstrated statistically significant improvement for participants in several key areas through multiple studies.
With thanks to Rett Syndrome Research Trust for arranging this wonderful interview with Professor Sir Adrian Bird describing his amazing breakthrough discovery of MECP2 and his ongoing scientific research.
An award presented to Distinguished Professor Margaret Brimble from the parents of Rett children.
"For your expert knowledge and years of personal and professional service to Rett in New Zealand. Thank you for your caring and commitment to helping Rett children, adults and their families."
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Send us an e-mail
Do you have any comments or questions about Rett syndrome? Please send us an e-mail at rett.info@nzord.org.nz.
Patron and Ambassador for Rett New Zealand
Photo credits: Glen Chean
Dame Margaret Brimble FRS is a Distinguished Professor and Director of Medicinal Chemistry at the University of Auckland. Margaret has graciously accepted the role of Patron and Ambassador for Rett New Zealand. Rett NZ is delighted that Margaret has taken on this position, and we are very grateful.
A visit to the Brimble Lab to see where Trofinetide (NNZ-2566) was discovered
Distinguished Professor Margaret Brimble of the University of Auckland has given two lectures at the Rett NZ Conferences.
In May 2016 Professor Brimble invited our Rett NZ Trust Chairman Dugald MacBrayne and Rett NZ Coordinator Gillian Deane to visit her laboratory to see how the drug NNZ-2566 or Trofinetide was discovered and to meet the staff members who helped with its discovery. There are no current approved medicines for the treatment of Rett Syndrome.
Julia Roberts became committed to helping find a cure for Rett Syndrome after meeting Abigail Brodsky, a young girl battling the disease. Julia appeared in the Discovery Channel's Silent Angels: The Rett Syndrome Story and testified before Congress about the need for more research on the crippling neurological disease.
Rose Hill Designs: Believe in a Cure
Illustration by Heather Stillufsen
