Clinical trial for Rett syndrome launched

Study marks the emergence of disease-modifying treatments for autism spectrum disorders

December 16, 2010

Boston, Mass. -- Researchers at Children's Hospital Boston have begun a randomized, placebo-controlled trial to test a potential drug treatment for Rett syndrome, the leading known genetic cause of autism in girls. The drug, mecasermin, a synthetic form of insulin-like growth factor-1 (IGF-1), is already FDA-approved for children with short stature due to IGF-1 deficiency. Read more

Rett Syndrome Video Channel: The Premier Place for Rett Syndrome Videos

Rett Syndrome Video Channel: The Premier Place for Rett Syndrome Videos

Researchers created autistic nerve cells

A multi-institution research project led by Alysson R. Muotri of the University of California, San Diego, has created a cellular model to aid study of a form of autism.

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