A pilot controlled trial of insulin-like growth factor-1 in children with Phelan-McDermid syndrome

Abstract

BACKGROUND Autism spectrum disorder (ASD) is now understood to have multiple genetic risk genes and one example is SHANK3. SHANK3 deletions and mutations disrupt synaptic function and result in Phelan-McDermid syndrome (PMS), which causes a monogenic form of ASD with a frequency of at least 0.5% of ASD cases. Recent evidence from preclinical studies with… (More)
DOI: 10.1186/2040-2392-5-54

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@inproceedings{Kolevzon2014APC, title={A pilot controlled trial of insulin-like growth factor-1 in children with Phelan-McDermid syndrome}, author={Alexander Kolevzon and L. A. M. Bush and A. Ting Wang and Danielle Halpern and Yitzchak Frank and David Grodberg and Robert Rapaport and Teresa Tavassoli and William Chaplin and Latha V. Soorya and Joseph D. Buxbaum}, booktitle={Molecular autism}, year={2014} }