Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial

Author:

Longo Nicola,Harding Cary O,Burton Barbara K,Grange Dorothy K,Vockley Jerry,Wasserstein Melissa,Rice Gregory M,Dorenbaum Alejandro,Neuenburg Jutta K,Musson Donald G,Gu Zhonghua,Sile Saba

Publisher

Elsevier BV

Subject

General Medicine

Reference16 articles.

1. Hyperphenylalaninemia: phenylalanine hydroxylase deficiency;Scriver,2001

2. Phenylalanine hydroxylase deficiency;Mitchell;Genet Med,2011

3. Inherited metabolic disorders in Turkey;Ozalp;J Inherit Metab Dis,1990

4. Molecular structure and polymorphic map of the human phenylalanine hydroxylase gene;DiLella;Biochemistry,1986

5. Long term follow-up of patients with inborn errors of metabolism detected by the newborn screening program in Japan;Aoki;Southeast Asian J Trop Med Public Health,2003

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