Promising news from International Rett Syndrome Foundation

The IRSF International Rett Syndrome Foundation <irsflash@rettsyndrome.org> announced today that it is funding a Phase 2b placebo-controlled, cross-over study of insulin-like growth factor-1 (IGF1) in a larger group of Rett syndrome patients. This decision indicates that the Phase 1 trial showed the treatment was safe and possibly effective. Earlier work in a mouse model demonstrated good efficacy of IGF treatment. The new trial involves injection IGF1 protein, rather than gene therapy. It will be conducted at Boston Children’s Hospital. Rett syndrome is a disease primarily affecting girls, who exhibit some of the features of autism. Full disclosure: I am on the Scientific Advisory Board of the IRSF. Questions regarding the trial can be directed to: Theresa.Trapilo@childrens.harvard.edu

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