Concepedia

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

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

The Lancet · 2014 · 167 citations · 15 references

DOI

Concepts

References

15

The metabolic and molecular bases of inherited disease

Ying-Tsong Chen · Medical Entomology and Zoology · 2001 · 6.9K citations

, , +14

How practical are recommendations for dietary control in phenylketonuria?

JH Walter, F. White, SK Hall et al. · The Lancet · 2002 · 301 citations

, , +8

Molecular structure and polymorphic map of the human phenylalanine hydroxylase gene

Anthony G. DiLella, Simon C.M. Kwok, Fred D. Ledley et al. · Biochemistry · 1986 · 173 citations

, , +11

Preclinical evaluation of multiple species of PEGylated recombinant phenylalanine ammonia lyase for the treatment of phenylketonuria

Christineh N. Sarkissian, Alejandra Gámez, Wang Lin et al. · Proceedings of the National Academy of Sciences · 2008 · 114 citations · Full text

, , +17

Executive function impairment in early‐treated PKU subjects with normal mental development

Vincenzo Leuzzi, M. Pansini, Enzo Sechi et al. · Journal of Inherited Metabolic Disease · 2004 · 104 citations

, , +16