Concepedia

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry

Neal J. Weinreb, Joel Charrow, Hans C. Andersson, Paige Kaplan, Edwin H. Kolodny, Pramod K. Mistry, Gregory M. Pastores, Barry E. Rosenbloom, C. Ronald Scott, Rebecca S. Wappner,

The American Journal of Medicine · 2002 · 541 citations · 32 references

DOI

Concepts

References

32

Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease

Norman W. Barton, Roscoe O. Brady, James M. Dambrosia et al. · New England Journal of Medicine · 1991 · 1.3K citations · Full text

, , +17

Metabolism of glucocerebrosides II. Evidence of an enzymatic deficiency in Gaucher's disease

Roscoe O. Brady, Julian N. Kanfer, David J. Shapiro · Biochemical and Biophysical Research Communications · 1965 · 805 citations

, , +14

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis

Timothy M. Cox, Robin Lachmann, C. E. Hollak et al. · The Lancet · 2000 · 744 citations

, , +4

The Gaucher Registry

Joel Charrow, Hans C. Andersson, Paige Kaplan et al. · Archives of Internal Medicine · 2000 · 585 citations

, , +17

Pulmonary hypertension complicating portal hypertension: Prevalence and relation to splanchnic hemodynamics

Antoine Hadengue, Mohamed Kamal Benhayoun, Didier Lebrec et al. · Gastroenterology · 1991 · 497 citations

, , +12