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

Gheona Altarescu, Mali Szleifer, Sharon Hashmueli and David Aviezer Heitner, Hanna Rosenbaum, Pilar Giraldo, Atul Mehta, Glen Park, Mici Phillips, Deborah Elstein, Terreros Muñoz, Sergio E. Solorio-Meza, Dominick Amato, Gloria Duran, Fiorina Giona, Rene Ari Zimran, Einat Brill-Almon, Raul Chertkoff, Milan Petakov, Francisco Blanco-Favela, Eduardo disease taliglucerase alfa, a novel enzyme replacement therapy for Gaucher expressed recombinant glucocerebrosidase, − Pivotal trial with plant cell

[1]  A. Haims,et al.  Focal splenic lesions in type I Gaucher disease are associated with poor platelet and splenic response to macrophage-targeted enzyme replacement therapy , 2010, Journal of Inherited Metabolic Disease.

[2]  J. Dambrosia,et al.  Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease. , 1991, The New England journal of medicine.

[3]  O. Dym,et al.  Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system. , 2007, Plant biotechnology journal.

[4]  D. Häussinger,et al.  Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: a 2-center retrospective analysis. , 2006, Blood.

[5]  J. Yee,et al.  A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase , 2008, American journal of hematology.

[6]  E. Levy-Lahad,et al.  Replacement therapy with imiglucerase for type 1 Gaucher's disease , 1995, The Lancet.

[7]  T. Cox Recommendations for treating patients with Gaucher disease with emerging enzyme products. , 2010, Blood cells, molecules & diseases.

[8]  G. Andria,et al.  The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: A position statement , 2003, Journal of Inherited Metabolic Disease.

[9]  J. Kaiser Is the Drought Over for Pharming? , 2008, Science.

[10]  E. Galun,et al.  A Plant-Derived Recombinant Human Glucocerebrosidase Enzyme—A Preclinical and Phase I Investigation , 2009, PloS one.

[11]  J. Yee,et al.  The long-term international safety experience of imiglucerase therapy for Gaucher disease. , 2007, Molecular genetics and metabolism.

[12]  G. Andria,et al.  Therapeutic goals in the treatment of Gaucher disease. , 2004, Seminars in hematology.

[13]  S. Packman,et al.  Phenotypic heterogeneity of N370S homozygotes with type I Gaucher disease: An analysis of 798 patients from the ICGG Gaucher Registry , 2008, Journal of Inherited Metabolic Disease.

[14]  M. Horowitz,et al.  Low-dose enzyme replacement therapy for Gaucher's disease: effects of age, sex, genotype, and clinical features on response to treatment. , 1994, The American journal of medicine.

[15]  G. D. den Heeten,et al.  Quantification of skeletal involvement in adults with type I Gaucher's disease: fat fraction measured by Dixon quantitative chemical shift imaging as a valid parameter. , 2002, AJR. American journal of roentgenology.

[16]  J. Karlawish,et al.  The continuing unethical conduct of underpowered clinical trials. , 2002, JAMA.

[17]  A. O'Hagan,et al.  The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review. , 2006, Health technology assessment.

[18]  B. Bembi,et al.  Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease. , 2010, Blood cells, molecules & diseases.

[19]  M. Mori,et al.  Dosing enzyme replacement therapy for Gaucher disease: older, but are we wiser? , 2009, Genetics in Medicine.

[20]  R. Steinbrook Drug shortages and public health. , 2009, The New England journal of medicine.

[21]  R. Schiffmann,et al.  Enzyme Therapy in Type 1 Gaucher Disease: Comparative Efficacy of Mannose-Terminated Glucocerebrosidase from Natural and Recombinant Sources , 1995, Annals of Internal Medicine.