70 Years of PKU Scientific Progress

> Basic Research on
Phenylketonuria (PKU)

> Therapeutic Development
Research on PKU

> PKU Clinical Development

> Stevens Lab Publications
on PKU

> Information on Genotype
Testing for PAH Mutations

< Back to PKU Research

Key Dates

1934 - Asborn Folling describes PKU

1935 - Lionel Penrose assigns the name PKU

1937 - George Jervis PKU due to enzyme deficiency

1953 - Horst Bickel Suggest dietary treatment

1961 - Robert Guthrie develops simple and inexpensive test for PKU

1966 - Guthrie test becomes standard

1967 - Dick Koch creates National Study on PKU

1992 - Savio Woo identifies gene PAH

1993 - Diet for life recommended McDonald Creates PKU mouse

1996 - Stevens Lab determines PAH 3D Structure

1999 - Kure/Blau propose BH4 treatment

2007 - Kuvan approved

2008 - PEG-PAL enters clinical trials

Total: 5624 Scientific Publications

Sapropterin (KUVAN TM) Status

RxTrials Institute Drug Pipeline Alert May 28, 2008  | Vol. 6 No. 22

BioMarin Initiates Phase I Clinical Study of PEG-PAL BioMarin Pharmaceutical has started treatment in its Phase I clinical study of PEG-PAL for phenylketonuria (PKU).

The primary objective of the study is to assess the safety and tolerability of single, subcutaneous injections of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) in subjects with PKU, the company said. The open-label, multicenter study will be conducted in roughly 35 PKU patients. BioMarin said it expects enrollment to be completed late this year.