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Filters applied: Meta-Analysis, Randomized Controlled Trial. Clear all
Page 1
Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.
Cox TM, Drelichman G, Cravo R, Balwani M, Burrow TA, Martins AM, Lukina E, Rosenbloom B, Ross L, Angell J, Puga AC. Cox TM, et al. Lancet. 2015 Jun 13;385(9985):2355-62. doi: 10.1016/S0140-6736(14)61841-9. Epub 2015 Mar 26. Lancet. 2015. PMID: 25819691 Clinical Trial.
FINDINGS: Between Sept 15, 2009, and Nov 9, 2011, we randomly allocated 106 (66%) patients to eliglustat and 54 (34%) to imiglucerase. In the per-protocol population, 84 (85%) of 99 patients who completed eliglustat treatment and 44 (94%) of 47 patients who completed im
FINDINGS: Between Sept 15, 2009, and Nov 9, 2011, we randomly allocated 106 (66%) patients to eliglustat and 54 (34%) to imiglucerase
Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease.
Ben Turkia H, Gonzalez DE, Barton NW, Zimran A, Kabra M, Lukina EA, Giraldo P, Kisinovsky I, Bavdekar A, Ben Dridi MF, Gupta N, Kishnani PS, Sureshkumar EK, Wang N, Crombez E, Bhirangi K, Mehta A. Ben Turkia H, et al. Am J Hematol. 2013 Mar;88(3):179-84. doi: 10.1002/ajh.23382. Epub 2013 Feb 9. Am J Hematol. 2013. PMID: 23400823 Free article. Clinical Trial.
This study compared the efficacy and safety of velaglucerase alfa with imiglucerase, the previous standard of care. A 9-month, global, randomized, double-blind, non-inferiority study compared velaglucerase alfa with imiglucerase (60 U/kg every other week) in treatme …
This study compared the efficacy and safety of velaglucerase alfa with imiglucerase, the previous standard of care. A 9-month, global …
A randomized trial comparing the efficacy and safety of imiglucerase (Cerezyme) infusions every 4 weeks versus every 2 weeks in the maintenance therapy of adult patients with Gaucher disease type 1.
Kishnani PS, DiRocco M, Kaplan P, Mehta A, Pastores GM, Smith SE, Puga AC, Lemay RM, Weinreb NJ. Kishnani PS, et al. Mol Genet Metab. 2009 Apr;96(4):164-70. doi: 10.1016/j.ymgme.2008.12.015. Epub 2009 Feb 4. Mol Genet Metab. 2009. PMID: 19195916 Clinical Trial.
Imiglucerase (Cerezyme) has been the standard of care for treatment of Gaucher disease, a lysosomal storage disorder resulting from deficiency of glucocerebrosidase, since its approval in 1994. ...This was a 24-month, open-label, randomized, Phase 4, dose-frequency study c
Imiglucerase (Cerezyme) has been the standard of care for treatment of Gaucher disease, a lysosomal storage disorder resulting from d
A Systematic Review and Meta-analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease.
Leonart LP, Fachi MM, Böger B, Silva MRD, Szpak R, Lombardi NF, Pedroso MLA, Pontarolo R. Leonart LP, et al. Ann Pharmacother. 2023 Mar;57(3):267-282. doi: 10.1177/10600280221108443. Epub 2022 Jul 11. Ann Pharmacother. 2023. PMID: 35815393
Following screening and eligibility assessment, 68 reports were included. The studies evaluated imiglucerase, velaglucerase alfa, taliglucerase alfa, miglustat, and eliglustat. The results showed that ERT is effective as a treatment in both naive and experienced patients. …
Following screening and eligibility assessment, 68 reports were included. The studies evaluated imiglucerase, velaglucerase alfa, tal …
A Randomized, Double-Blind, 2-Treatment, 2-Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration.
Kang E, Kim D, Hwang S, Lemech C, Wharton J, Lee Y, Yoo HW, Lee BH. Kang E, et al. Mol Genet Genomic Med. 2025 Jun;13(6):e70111. doi: 10.1002/mgg3.70111. Mol Genet Genomic Med. 2025. PMID: 40515481 Free PMC article. Clinical Trial.
BACKGROUND: Imiglucerase (Cerezyme; Sanofi, Paris, France), an analogue of beta-glucocerebrosidase produced by recombinant DNA technology, has been a safe and effective treatment for Gaucher disease (GD) for over 25 years. A new imiglucerase, Abcertin (Seongnam-si, …
BACKGROUND: Imiglucerase (Cerezyme; Sanofi, Paris, France), an analogue of beta-glucocerebrosidase produced by recombinant DNA techno …
Enzyme replacement and substrate reduction therapy for Gaucher disease.
Shemesh E, Deroma L, Bembi B, Deegan P, Hollak C, Weinreb NJ, Cox TM. Shemesh E, et al. Cochrane Database Syst Rev. 2015 Mar 27;2015(3):CD010324. doi: 10.1002/14651858.CD010324.pub2. Cochrane Database Syst Rev. 2015. PMID: 25812601 Free PMC article.
One study examined substrate reduction therapy in people with chronic neuronopathic (type 3) Gaucher disease who continued to receive enzyme replacement therapy.Treatment-naive participants had similar increases in haemoglobin when comparing those receiving imiglucerase or …
One study examined substrate reduction therapy in people with chronic neuronopathic (type 3) Gaucher disease who continued to receive enzyme …
Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.
Cox TM, Drelichman G, Cravo R, Balwani M, Burrow TA, Martins AM, Lukina E, Rosenbloom B, Goker-Alpan O, Watman N, El-Beshlawy A, Kishnani PS, Pedroso ML, Gaemers SJM, Tayag R, Peterschmitt MJ. Cox TM, et al. Blood. 2017 Apr 27;129(17):2375-2383. doi: 10.1182/blood-2016-12-758409. Epub 2017 Feb 6. Blood. 2017. PMID: 28167660 Free PMC article. Clinical Trial.
In the phase 3 Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE), at 1 year, eliglustat was noninferior to imiglucerase enzyme therapy in maintaining stable platelet count …
In the phase 3 Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Re …
Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.
Elstein D, Dweck A, Attias D, Hadas-Halpern I, Zevin S, Altarescu G, Aerts JF, van Weely S, Zimran A. Elstein D, et al. Blood. 2007 Oct 1;110(7):2296-301. doi: 10.1182/blood-2007-02-075960. Epub 2007 Jul 3. Blood. 2007. PMID: 17609429 Free article. Clinical Trial.
Enzyme replacement therapy (ERT) with imiglucerase reduces hepatosplenomegaly and improves hematologic parameters in Gaucher disease type 1 within 6-24 months. ...Mean change in platelet counts was only significant between miglustat and imiglucerase groups (P = .035 …
Enzyme replacement therapy (ERT) with imiglucerase reduces hepatosplenomegaly and improves hematologic parameters in Gaucher disease …
Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources.
Grabowski GA, Barton NW, Pastores G, Dambrosia JM, Banerjee TK, McKee MA, Parker C, Schiffmann R, Hill SC, Brady RO. Grabowski GA, et al. Ann Intern Med. 1995 Jan 1;122(1):33-9. doi: 10.7326/0003-4819-122-1-199501010-00005. Ann Intern Med. 1995. PMID: 7985893 Clinical Trial.
OBJECTIVE: To compare the efficacy of mannose-terminated glucocerbrosidase prepared from natural (alglucerase; Ceredase, Genzyme Corp., Cambridge, Massachusetts) and recombinant (imiglucerase; Cerezyme, Genzyme Corp.) sources in treating type 1 Gaucher disease. DESIGN: Dou …
OBJECTIVE: To compare the efficacy of mannose-terminated glucocerbrosidase prepared from natural (alglucerase; Ceredase, Genzyme Corp., Camb …