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Settings standards for research into Rett syndrome
Date:10/31/2012

l Rett Syndrome Foundation (IRSF) and the Rett Syndrome Research Trust (RSRT). Workshop participants included key members of the Rett syndrome research community, including basic scientists, clinicians, and representatives from the National Institutes of Health (NIH), the Food and Drug Administration (FDA), the pharmaceutical industry and private foundations. Several workshop participants contributed to the article, and the corresponding authors are Dr. David Katz (Case Western Reserve University School of Medicine), Dr. Laura Mamounas (National Institute of Neurological Disorders and Stroke) and Dr. Huda Zoghbi (Baylor College of Medicine).

The main aim of the workshop was to identify crucial knowledge gaps in Rett syndrome research at the preclinical level, and to suggest scientific priorities and best practices for the use of animal models in preclinical evaluation of potential new therapeutics. As explained in the new review article, the combination of an urgent need for effective treatments for Rett syndrome, coupled with the availability of good mouse models, is a driving force for studies that can identify and test new drugs. The outcome of the workshop included a set of recommended guidelines for animal studies of Rett syndrome, to ensure that decisions to initiate costly clinical trials will be founded on reliable data that is produced using standardized study design and transparent reporting. Ultimately, the increased level of rigor in animal studies should shorten the time to effective treatments.


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Contact: Sarah E. Allan, PhD
sarah.allan@biologists.com
International Rett Syndrome Foundation
Source:Eurekalert

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