Rett Syndrome Pediatric Research and Clinical Trials

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Contact the Rett Syndrome Program

Research into the history of Rett syndrome

Because better medical care is allowing many people with Rett syndrome to live longer, there is a lot that needs to be learned about how the disease progresses, what the quality of life is for patients and what we can do to further improve care.

Boston Children’s is one of four institutions working together to study the natural history of Rett syndrome through the Rare Diseases Clinical Research Network. Researchers from Boston Children's Hospital travel around the United States to evaluate children and adults as part of this study. The study is funded by the National Institutes of Health, including its Office of Rare Diseases Research.

Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation with Recombinant Human IGF-1 (mecasermin [rDNA] injection)

Principal Investigator: Mustafa Sahin, MD, PhD

For information about the IGF-1 trial:


Autism Consortium

Boston Children’s is part of the Autism Consortium, a group of researchers, clinicians and families dedicated to accelerating research and enhancing clinical care for autism and related disorders including Rett syndrome.
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- Sandra L. Fenwick, President and CEO

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