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.
D045930:Anabolic Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D008297:Male; D010649:Phenylalanine / Q000097:blood; D010661:Phenylketonurias / Q000097:blood / Q000188:drug therapy*; D013197:Stanozolol / Q000627:therapeutic use; D014443:Tyrosine / Q000097:blood
D045930:Anabolic Agents; D014443:Tyrosine; D010649:Phenylalanine; D013197:Stanozolol