Citations (36)
Keep up to date with the latest research on this topic with citation updates for this article.
Read on this site (6)
Saptashwa Datta, K Narayanan Rajnish, C George Priya Doss, S. Melvin Samuel, E. Selvarajan & Hatem Zayed. (2020) Enzyme therapy: a forerunner in catalyzing a healthy society?. Expert Opinion on Biological Therapy 20:10, pages 1151-1174.
Read now
Read now
Christine Serratrice, Sebastian Carballo, Jacques Serratrice & Jérome Stirnemann. (2016) Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy. Core Evidence 11, pages 37-47.
Read now
Read now
Barry E Rosenbloom & Neal J Weinreb. (2014) Bone disease in patients with Gaucher disease. Expert Review of Endocrinology & Metabolism 9:2, pages 153-162.
Read now
Read now
Thomas Kirkegaard. (2013) Emerging therapies and therapeutic concepts for lysosomal storage diseases. Expert Opinion on Orphan Drugs 1:5, pages 385-404.
Read now
Read now
Michael Beck. (2010) Emerging drugs for lysosomal storage diseases. Expert Opinion on Emerging Drugs 15:3, pages 495-507.
Read now
Read now
Carla EM Hollak, Maaike de Fost, Laura van Dussen, Stephan vom Dahl & Johannes MFG Aerts. (2009) Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose – response relationships. Expert Opinion on Pharmacotherapy 10:16, pages 2641-2652.
Read now
Read now
Articles from other publishers (30)
Neal J. Weinreb, Ozlem Goker-Alpan, Priya S. Kishnani, Nicola Longo, T. Andrew Burrow, John A. Bernat, Punita Gupta, Nadene Henderson, Helio Pedro, Carlos E. Prada, Divya Vats, Ravi R. Pathak, Ekaterina Wright & Can Ficicioglu. (2022) The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?. Molecular Genetics and Metabolism 136:1, pages 4-21.
Crossref
Crossref
Bharti Sapra, Dhriti Mahajan, Sagar Chaudhary & Ashok Kumar Tiwary. 2022. Drug Delivery Systems for Metabolic Disorders. Drug Delivery Systems for Metabolic Disorders
371
409
.
Benedita Kaç Labbé Feron & Simon Clifford Wainwright Richardson. (2019)
The Delivery of Personalised, Precision Medicines
via
Synthetic Proteins
. Drug Delivery Letters 9:2, pages 79-88.
Crossref
Crossref
Mary Anne D. Chiong, Marie Julianne C. Racoma & Mary Ann R. Abacan. (2018) Genetic and clinical characteristics of Filipino patients with Gaucher disease. Molecular Genetics and Metabolism Reports 15, pages 110-115.
Crossref
Crossref
Amal El-Beshlawy, Anna Tylki-Szymanska, Ashok Vellodi, Nadia Belmatoug, Gregory A. Grabowski, Edwin H. Kolodny, Julie L. Batista, Gerald F. Cox & Pramod K. Mistry. (2017) Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry. Molecular Genetics and Metabolism 120:1-2, pages 47-56.
Crossref
Crossref
Jiajia Wang, Xiaomin Wang, Yunyan Zhao, Xiaoyao Ma, Yue Wan, Zhongwei Chen, Hao Chen, Hao Gan, Jing Li, Lei Li, Peng George Wang & Wei Zhao. (2016)
Synthesis and biological evaluation of
d
-gluconhydroximo-1,5-lactam and its oxime-substituted derivatives as pharmacological chaperones for the treatment of Gaucher disease
. MedChemComm 7:2, pages 365-370.
Crossref
Crossref
Geertje M. de Boer, Laura van Dussen, Leon M. van den Toorn, Michael A. den Bakker, Rogier A.S. Hoek, Dennis A. Hesselink, Carla E.M. Hollak & Peter Th. W. van Hal. (2016) Lung Transplantation in Gaucher Disease. Chest 149:1, pages e1-e5.
Crossref
Crossref
Jérôme Stirnemann, Christian Rose, Christine Serratrice, Florence Dalbies, Olivier Lidove, Agathe Masseau, Yves-Marie Pers, Camille Baron & Nadia Belmatoug. (2015) Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1. Orphanet Journal of Rare Diseases 10:1.
Crossref
Crossref
Mannching Sherry Ku. (2015) Recent trends in specialty pharma business model. Journal of Food and Drug Analysis 23:4, pages 595-608.
Crossref
Crossref
Carla E.M. Hollak & Neal J. Weinreb. (2015) The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease. Best Practice & Research Clinical Endocrinology & Metabolism 29:2, pages 205-218.
Crossref
Crossref
Simon A Smith, Tim French & Simon J Hollingsworth. (2014) The impact of germline mutations on targeted therapy. The Journal of Pathology 232:2, pages 230-243.
Crossref
Crossref
Gregory A GrabowskiBarry E Rosenbloom & Neal J Weinreb. 2013. Gaucher Disease: Basic and Clinical Perspectives. Gaucher Disease: Basic and Clinical Perspectives
26
49
.
Peiqing Zhang, Tianhua Wang, Muriel Bardor & Zhiwei Song. (2013) Deciphering O-glycomics for the development and production of biopharmaceuticals. Pharmaceutical Bioprocessing 1:1, pages 89-104.
Crossref
Crossref
Ana Trapero & Amadeu Llebaria. (2013) Glucocerebrosidase inhibitors for the treatment of Gaucher disease. Future Medicinal Chemistry 5:5, pages 573-590.
Crossref
Crossref
Neal J. Weinreb & Barry E. Rosenbloom. (2013) Splenomegaly, hypersplenism, and hereditary disorders with splenomegaly. Open Journal of Genetics 03:01, pages 24-43.
Crossref
Crossref
Ana Trapero, Meritxell Egido-Gabás & Amadeu Llebaria. (2013) Adamantane substituted aminocyclitols as pharmacological chaperones for Gaucher disease. MedChemComm 4:12, pages 1584.
Crossref
Crossref
Nico Lingg, Peiqing Zhang, Zhiwei Song & Muriel Bardor. (2012) The sweet tooth of biopharmaceuticals: Importance of recombinant protein glycosylation analysis. Biotechnology Journal 7:12, pages 1462-1472.
Crossref
Crossref
V. Valayannopoulos, A. Brassier, A. Chabli, C. Caillaud, M. Lemoine, T. Odent, J.B. Arnoux & P. de Lonlay. (2011) Le traitement par enzymothérapie des maladies lysosomales. Archives de Pédiatrie 18:10, pages 1119-1123.
Crossref
Crossref
Boris Brumshtein, Matilde Aguilar-Moncayo, Juan M. Benito, José M. García Fernandez, Israel Silman, Yoseph Shaaltiel, David Aviezer, Joel L. Sussman, Anthony H. Futerman & Carmen Ortiz Mellet. (2011) Cyclodextrin-mediated crystallization of acid β-glucosidase in complex with amphiphilic bicyclic nojirimycin analogues. Organic & Biomolecular Chemistry 9:11, pages 4160.
Crossref
Crossref
Pramod K. Mistry, Neal J. Weinreb, Paige Kaplan, J. Alexander Cole, Andrea R. Gwosdow & Thomas Hangartner. (2011) Osteopenia in Gaucher disease develops early in life: Response to imiglucerase enzyme therapy in children, adolescents and adults. Blood Cells, Molecules, and Diseases 46:1, pages 66-72.
Crossref
Crossref
M Rajappa, A Goyal & J Kaur. (2009) Inherited metabolic disorders involving the eye: a clinico-biochemical perspective. Eye 24:4, pages 507-518.
Crossref
Crossref
Dominique P. Germain, Patrick Bruneval, Thi-Chien Tran, Pierre Balouet, Bernard Richalet & Karelle Benistan. (2010) Uneventful pregnancy outcome after enzyme replacement therapy with agalsidase beta in a heterozygous female with Fabry disease: A case report. European Journal of Medical Genetics 53:2, pages 111-112.
Crossref
Crossref
Gregory M. Pastores. (2010) Recombinant Glucocerebrosidase (Imiglucerase) as a Therapy for Gaucher Disease. BioDrugs 24:1, pages 41-47.
Crossref
Crossref
Michel Leporrier. (2009) Maladie de Gaucher de type 1 et grossesse. La Presse Médicale 38, pages 2S58-2S60.
Crossref
Crossref
Thierry Schaeverbeke. (2009) Conséquences ostéo-articulaires de la maladie de Gaucher et efficacité thérapeutique de l’imiglucérase sur la masse osseuse chez l’adulte. Revue de la littérature. La Presse Médicale 38, pages 2S38-2S41.
Crossref
Crossref
Tse Siang Kang & Raymond C. Stevens. (2009) Structural aspects of therapeutic enzymes to treat metabolic disorders. Human Mutation 30:12, pages 1591-1610.
Crossref
Crossref
Neal J. Weinreb. (2009) Mesenchymal gaucho homing on the range. Blood 114:15, pages 3134-3135.
Crossref
Crossref
Yaron Ilan, Deborah Elstein & Ari Zimran. (2009) Glucocerebroside: an evolutionary advantage for patients with Gaucher disease and a new immunomodulatory agent. Immunology & Cell Biology 87:7, pages 514-524.
Crossref
Crossref
David Aviezer, Einat Brill-Almon, Yoseph Shaaltiel, Sharon Hashmueli, Daniel Bartfeld, Sarah Mizrachi, Yael Liberman, Arnold Freeman, Ari Zimran & Eithan Galun. (2009) A Plant-Derived Recombinant Human Glucocerebrosidase Enzyme—A Preclinical and Phase I Investigation. PLoS ONE 4:3, pages e4792.
Crossref
Crossref
Michael Beck. (2009) Therapy for lysosomal storage disorders. IUBMB Life, pages n/a-n/a.
Crossref
Crossref