Neuren granted Orphan Drug Status for NNZ-2566

Thank you to our donors and families, as well as Cure Rett for their continued partnership in helping to make this possible!

Press Release Press Release Orphan Drug Status: Explained Orphan Drug Status: Explained

We are in the Time of Rett

Our Research to Reality campaign aims to find a treatment and a cure for Rett syndrome.

Donate today Donate today Research to Reality Research to Reality

Neuren Announces Results of the NNZ-2566 Clinical Trial for Rett Syndrome

Read the Press Release Read the Press Release

Agreement announced to fund research for Natural History Study

Research Consortium, to be led by Dr. Alan Percy, University of Alabama at Birmingham

Rettsyndrome.org is the world's leading Rett syndrome research funding organization

Our impact Our impact Join Rettsyndrome.org Join Rettsyndrome.org

We have invested $35 million in Rett syndrome research

We’re leading the race to find effective treatments and a cure for Rett.

Research opportunities Research awards Research opportunities Research awards

We are funding successful clinical trials

We are conducting adult and pediatric clinical trials at top research universities including Harvard and Baylor. In 2015, we will begin Stage 3 clinical trails to prove drug efficacy.

Research strategy Clinical trials Research strategy Clinical trials

Our research will help millions

A cure for Rett will yield major advances toward treatments for millions of people around the world with Autism, Parkinson's, Alzheimers, Schizophrenia and Traumatic Brain Injuries.

Make a gift online Make a gift online

News

Neuren and the Orphan Drug Status: An Overview

February 18, 2015

This week’s news that Neuren Pharmaceuticals has been granted Orphan Drug Status by the FDA for NNZ-2566, now known as Trofinetide, is good news for families who live with Rett Syndrome.

IEPs and School Placement: Exploring Perspectives Empowers the Parent

December 17, 2014

An IEP is HUGE! The IEP dictates our life at the school, and will influence your child's life after high school. But . . . you probably already know how HUGE an IEP is. So, why should you spend your extremely precious and limited time listening to this presentation? Because it's powerful!

Rettsyndrome.org Talks About Advancing Research

December 15, 2014

Talk about how we choose a research project to fund and what exactly happens behind the scenes to ensure the Research Investments of Rettsyndrome.org are good ones.

See all news