Concepedia

Enzyme Replacement Therapy in a Patient with Gaucher Disease Type III: A Paradigmatic Case Showing Severe Adverse Reactions Started a Long Time After the Beginning of Treatment

Filippo Pinto e Vairo, Cristina Brinckmann Oliveira Netto, Alicia Dorneles, Suzana Mittelstadt, Matheus Vernet Machado Bressan Wilke, Divair Doneda, Kristiane Michelin, Camila Blos Ribeiro, Amanda Quevedo, Tatiane Alves Vieira,

JIMD Reports · 2013 · 12 citations · 19 references

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References

19

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 · 740 citations

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Prevention of Lysosomal Storage in Tay-Sachs Mice Treated with <i>N</i> -Butyldeoxynojirimycin

Frances M. Platt, Gabrielle R. Neises, Gabriele Reinkensmeier et al. · Science · 1997 · 342 citations

Pivotal trial with plant cell–expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease

Ari Zimran, Einat Brill‐Almon, Raul Chertkoff et al. · Blood · 2011 · 202 citations · Full text

A reappraisal of Gaucher disease—diagnosis and disease management algorithms

Pramod K. Mistry, Maria Domenica Cappellini, Elena Lukina et al. · American Journal of Hematology · 2010 · 187 citations

Consequences of diagnostic delays in type 1 Gaucher disease: The need for greater awareness among Hematologists–Oncologists and an opportunity for early diagnosis and intervention

Pramod K. Mistry, Sara Sadan, Ruhua Yang et al. · American Journal of Hematology · 2007 · 167 citations