Background: Rett syndrome is a progressive neurodevelopmental disorder, affecting approximately 1 in 10,000 girls. Be sure to wear …, Cincinnati Children’s Hospital Rett Clinic Town Hall Meeting, presented by Rettsyndrome.org Date: Sat, Oct 17, 2020 Time: 12:00 PM – 1:30 PM EDT Are you having difficulty understanding the current state of clinical care recommendations …, 18th Annual SoCal Virtual Picnic & Strollathon Join us for the 18th Annual SoCal Picnic & Strollathon – Virtual Edition! Dr. Fugere has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of AveXis, a Novartis company. Stay timely. Recent studies using rodent models demonstrated that re-expression of MeCP2 ameliorates Rett-syndrome-like phenotypes. Dr. Huffenberger has nothing to disclose. It also reaffirmed its commitment “to the Rett program and to ensuring the highest levels of transparency and integrity with the patients and providers we serve, and health agencies.”. Email: [email protected] Therefore, a gene replacement therapy is a promising therapeutic strategy for this disease. Dr. Pichard is Rettsyndrome.org’s Chief Science Officer. In a statement to the Rett syndrome community, the Novartis-owned company said this decision was based on concerns with animal data given to the the U.S. Food and Drug Administration (FDA) to support its approval of Zolgensma (onasemnogene abeparvovec-xioi) for children with spinal muscular atrophy (SMA) up to age 2. To assist our loved ones as soon as possible, we at Rettsyndrome.org will continue seeking treatments to alleviate symptoms and improve quality of life while we wait for this research to help create a world without Rett syndrome. 3 W Garden St We are sorry that this post was not useful for you! We use cookies to ensure that we give you the best experience on our website. News Gene Therapy Takes Center Stage at 2019 NORD Summit . AveXis is postponing plans to open a clinical trial of its Rett syndrome gene therapy candidate AVXS-201.. “Out of caution,” it will instead conduct additional preclinical tests and quality controls checks before asking for new regulatory approval for a study in patients. Objective: Develop a gene replacement therapy for the treatment of Rett Syndrome. Dr. Dennys-Rivers has nothing to disclose. The first, which would deliver a functional MECP2 gene to cells, was supposed to go to trial in 2019. Dr. Miranda has nothing to disclose. Questions and comments submitted Dr. Kyle Fink and Dr. Dominique Pichard explore the use of CRISPR/Cas9 technologies for gene therapy in Rett syndrome research. Submitted comments are subject to editing and editor review prior to posting. Your organization or institution (if applicable), e.g. News Sarepta and StrideBio Partner to Advance Gene Therapies for Rett Syndrome, Other Disorders Dr. Thomsen has received research support from AveXis, a Novartis company. Mice were injected intracerebroventricularly with multiple doses, and behavior (e.g., rotarod and open field) and survival were monitored.

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