Methods and compositions for treating phenylketonuria

A - Human Necessities – 61 – K

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A61K 31/195 (2006.01) C12N 9/00 (2006.01)

Patent

CA 2691623

Provided herein are methods for treating a subject suffering from phenylketonuria by administering a phenylalanine hydroxylase ("PAH") and/or a phenylalanine ammonia lyase ("PAL") to the subject under conditions effective to deliver the pheny-lalanine 4-hydroxylase and/or PAL to the subject's small intestine. Also provided are methods for increasing the therapeutic activity of a phenylalanine 4-hydroxylase by thiolating the phenylalanine 4- hydroxylase. In addition, provided are oral dosage forms that include a phenylalanine 4-hydroxylase and/or a PAL and an enteric coating.

Méthodes de traitement d'un sujet souffrant de phénylcétonurie au moyen de phénylalanine hydroxylase ("PAH") et/ou de phénylalanine ammonia lyase ("PAL") dans des conditions efficaces pour l'administration de la phénylalanine 4-hydroxylase et/ou de PAL dans l'intestin grêle dudit sujet. L'invention concerne également des méthodes propres à accroître l'activité thérapeutique de la phénylalanine 4-hydroxylase par thiolation de cette dernière. Sont également décrites des formes racémiques orales comprenant une phénylalanine 4-hydroxylase et/ou PAL et un enrobage gastrorésistant.

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