Rett Syndrome Awareness Month has been incredible. As a community, you rallied like never before – speaking out to share the good, the bad, and the ugly about Rett syndrome to educate the world and move the needle. Your efforts continue to make a difference. Today, more people know about Rett syndrome than ever before … Read More
Dear Rett Community, Today we took a significant step forward with the trofinetide clinical development program initiating the pivotal Phase 3 LAVENDER study. As we have shared before, LAVENDER is a 12-week study that will evaluate the efficacy and safety of trofinetide versus placebo in approximately 180 girls and young women aged 5 to 20 … Read More
The International Rett Syndrome Foundation (doing business as Rettsyndrome.org) has officially welcomed Dominique Pichard, MD to the organization as their new Chief Science Officer. A highly accomplished clinician and researcher, Dr. Dominique Pichard brings years of hands-on experience conducting critical work in clinical trials with the National Institutes of Health (NIH), pharmaceutical companies, and the … Read More
Rettsyndrome.org Funds Research to Provide Hope for Females and Males Affected by MECP2 Mutation. CINCINNATI, OH – June 3 – Rettsyndrome.org announced their funding of two new research projects today. Jeannie T. Lee, MD, Ph.D. of Massachusetts General Hospital is awarded a two year ANGEL Grant for $600,000 to focus on reactivating the silent X … Read More
Rettsyndrome.org hosted a clinical Learning Collaborative on May 8 and 9 in Denver. The Learning Collaborative is made up of physicians and clinicians from Rett clinics around the country who volunteered their time to share ideas in the spirit of advancing care for Rett families, and to create a universal standard of excellence across all … Read More