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Deborah Elstein

Showing results (141-150 of 154) with videos related to

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American Journal of Hematology|March 18, 2015
Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher diseaseDeborah Elstein, Atul Mehta, Derralynn A Hughes, et al.
American Journal of Hematology|November 2, 2017
Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS)Ari Zimran, Nadia Belmatoug, Bruno Bembi, et al.
American Journal of Hematology|February 7, 2013
Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 studyDerlis E Gonzalez, Hadhami Ben Turkia, Elena A Lukina, et al.
Molecular Genetics and Metabolism|December 28, 2010
Pyrimethamine increases β-hexosaminidase A activity in patients with Late Onset Tay SachsEtty Osher, Aviva Fattal-Valevski, Liora Sagie, et al.
American Journal of Hematology|March 25, 2015
Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trialsDerralynn A Hughes, Derlis E Gonzalez, Elena A Lukina, et al.
Plos One|November 27, 2013
Glucosylsphingosine is a highly sensitive and specific biomarker for primary diagnostic and follow-up monitoring in Gaucher disease in a non-Jewish, Caucasian cohort of Gaucher disease patientsArndt Rolfs, Anne-Katrin Giese, Ulrike Grittner, et al.
JAMA Neurology|April 24, 2014
Comparison of Parkinson risk in Ashkenazi Jewish patients with Gaucher disease and GBA heterozygotesRoy N Alcalay, Tama Dinur, Timothy Quinn, et al.
Orphanet Journal of Rare Diseases|January 7, 2022
Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trialsDeborah Elstein, Nadia Belmatoug, Patrick Deegan, et al.
Orphanet Journal of Rare Diseases|September 28, 2013
Functional and genetic characterization of the non-lysosomal glucosylceramidase 2 as a modifier for Gaucher diseaseYildiz Yildiz, Per Hoffmann, Stefan Vom Dahl, et al.
Journal of Clinical Medicine|June 27, 2024
Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient RegistryDeborah Elstein, Nadia Belmatoug, Bruno Bembi, et al.
Pageof 16

Showing results (141-150 of 154) with videos related to

Sort By:
Pageof 16
American Journal of Hematology|March 18, 2015
Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher diseaseDeborah Elstein, Atul Mehta, Derralynn A Hughes, et al.
American Journal of Hematology|November 2, 2017
Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS)Ari Zimran, Nadia Belmatoug, Bruno Bembi, et al.
American Journal of Hematology|February 7, 2013
Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 studyDerlis E Gonzalez, Hadhami Ben Turkia, Elena A Lukina, et al.
Molecular Genetics and Metabolism|December 28, 2010
Pyrimethamine increases β-hexosaminidase A activity in patients with Late Onset Tay SachsEtty Osher, Aviva Fattal-Valevski, Liora Sagie, et al.
American Journal of Hematology|March 25, 2015
Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trialsDerralynn A Hughes, Derlis E Gonzalez, Elena A Lukina, et al.
Plos One|November 27, 2013
Glucosylsphingosine is a highly sensitive and specific biomarker for primary diagnostic and follow-up monitoring in Gaucher disease in a non-Jewish, Caucasian cohort of Gaucher disease patientsArndt Rolfs, Anne-Katrin Giese, Ulrike Grittner, et al.
JAMA Neurology|April 24, 2014
Comparison of Parkinson risk in Ashkenazi Jewish patients with Gaucher disease and GBA heterozygotesRoy N Alcalay, Tama Dinur, Timothy Quinn, et al.
Orphanet Journal of Rare Diseases|January 7, 2022
Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trialsDeborah Elstein, Nadia Belmatoug, Patrick Deegan, et al.
Orphanet Journal of Rare Diseases|September 28, 2013
Functional and genetic characterization of the non-lysosomal glucosylceramidase 2 as a modifier for Gaucher diseaseYildiz Yildiz, Per Hoffmann, Stefan Vom Dahl, et al.
Journal of Clinical Medicine|June 27, 2024
Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient RegistryDeborah Elstein, Nadia Belmatoug, Bruno Bembi, et al.
Pageof 16