The Rett Syndrome Program at Children’s Hospital Boston is the only specialist Rett syndrome program in New England. CHB sees more than 250 patients who have Rett syndrome and related disorders.
The physical symptoms of Rett syndrome, such as seizures, gastrointestinal problems and musculoskeletal problems, can often be alleviated and managed. The Rett Program brings together Children's physicians and therapists who are experienced in caring for children and adults with Rett syndrome.
David Urion, MD, PhD, Director
Rett Syndrome Program
Children's Hospital Boston/Harvard Medical School
300 Longwood Avenue
Boston, MA 02115
Researchers at Children's Hospital Boston are continuing the randomized, placebo-controlled trial to test a potential drug treatment for Rett syndrome. The drug, Mecasermin, a synthetic form of insulin-like growth factor-1 (IGF-1), was shown to be safe during the phase 1 trial.
The trial follows research in animal models which suggested that raising IGF-1 levels can reverse features of Rett syndrome by enhancing maturation of synapses -- the points of communication between brain cells.
For additional information on the Phase 2 trial, visit http://www.rettsyndrome.org/news-and-media/irsf-communications/press-releases/phase-2-clinical-trial-study-of-igf-1 or contact the Rett Syndrome Program at Children's Hospital Boston.
Other Clinical Trials
To learn more about current and past clinical trials, please visit http://www.rsrt.org/research/clinical-trialsstudies/