Phelan-McDermid Syndrome: Insights into Synaptic Pathology and Novel Therapeutics in ASD

ID Number 11-1555

Principal Investigator(s)
Alexander Kolevzon

Department(s) or Division(s)


The purpose of this study is to pilot the use of Insulin-Like Growth Factor-1 (IGF-1) treatment in Phelan-McDermid Syndrome in order to evaluate safety, tolerability, and efficacy for a core deficit of autism - social impairment. IGF-1 is an injection under the skin that contains human IGF-1. IGF-1 is approved by the FDA under the brand name Increlex for the treatment of children with short stature due to primary IGF-1 deficiency. It is being used off-label in the current study and is not FDA approved, nor has it yet been studied in humans for the treatment of Phelan-McDermid Syndrome.

Contact Information
Lauren Bush
(212) 241-2826

Recruiting Patients: Yes