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Although not evaluated for the prevention of autism in women with DBH deficiency or treatment of autism in children with genetic DBH deficiency, a clinical trial of the drug to treat DBH deficient mothers and children in order to prevent the development of autism would seem to be worthwhile after more extensive animal experiments are conducted to establish safety

doi: 10.1111/j.1600-065X.2010.00926.x 2 McLaneLMAbdel-HakeemMSWherryEJ

Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment