Prospects for a pharmacological treatment of phenylketonuria

The present study deals with a new tentative therapeutic approach of phenylketonuria, based on the use of Anabolic Steroids. Data have been obtained in 10 children, 3 females and 7 males, aged 4-12 years, all presenting the stigmata of true phenylketonuria. The Anabolic Steroids used were Norandrostenolone Phenylpropionate, administered intramuscularly, in increasing doses from 25 to 75 mg.a.d., for 2 years. The 3 older patients were always on a totally free diet, the 7 younger ones on a semi-free diet. The results obtained demonstrate a total normalization of the blood levels of phenylalanine and tyrosine all along the treatment. Moreover, improvements of the psychoneurological disorders were observed.