Concepedia

Long-Term Treatment with Sodium Phenylbutyrate in Ornithine Transcarbamylase-Deficient Patients

Alberto Burlina, H Ogier, H Korall, Friedrich K. Trefz

Molecular Genetics and Metabolism · 2001 · 61 citations · 13 references

DOI

Concepts

References

13

Urea cycle enzymes

Sw Brusilow · Medical Entomology and Zoology · 2001 · 347 citations

A Pilot Clinical Trial of Oral Sodium 4-Phenylbutyrate (Buphenyl) in Δ F508-Homozygous Cystic Fibrosis Patients: Partial Restoration of Nasal Epithelial CFTR Function

Ronald C. Rubenstein, Pamela L. Zeitlin · American Journal of Respiratory and Critical Care Medicine · 1998 · 339 citations

, , +7

Sodium 4-phenylbutyrate downregulates Hsc70: implications for intracellular trafficking of ΔF508-CFTR

Ronald C. Rubenstein, Pamela L. Zeitlin · American Journal of Physiology-Cell Physiology · 2000 · 265 citations

, , +15

Long-Term Treatment of Girls with Ornithine Transcarbamylase Deficiency

Nancy E. Maestri, Saul W. Brusilow, David B. Clissold et al. · New England Journal of Medicine · 1996 · 259 citations · Full text

, , +17

Gene redundancy and pharmacological gene therapy: Implications for X-linked adrenoleukodystrophy

Stephan Kemp, He-Ming Wei, Jyh‐Feng Lu et al. · Nature Medicine · 1998 · 250 citations

, , +9