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Year Number of Results
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20 results

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Filters applied: Meta-Analysis, Randomized Controlled Trial. Clear all
Page 1
Agalsidase-beta therapy for advanced Fabry disease: a randomized trial.
Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, Finkel R, Packman S, Bichet DG, Warnock DG, Desnick RJ; Fabry Disease Clinical Trial Study Group. Banikazemi M, et al. Ann Intern Med. 2007 Jan 16;146(2):77-86. doi: 10.7326/0003-4819-146-2-200701160-00148. Epub 2006 Dec 18. Ann Intern Med. 2007. PMID: 17179052 Free article. Clinical Trial.
INTERVENTION: Intravenous infusion of agalsidase beta (1 mg per kg of body weight) or placebo every 2 weeks for up to 35 months (median, 18.5 months). ...RESULTS: Thirteen (42%) of the 31 patients in the placebo group and 14 (27%) of the 51 patients in the agalsi …
INTERVENTION: Intravenous infusion of agalsidase beta (1 mg per kg of body weight) or placebo every 2 weeks for up to 35 month …
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tøndel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Wallace EL, et al. J Med Genet. 2024 May 21;61(6):520-530. doi: 10.1136/jmg-2023-109445. J Med Genet. 2024. PMID: 37940383 Free PMC article. Clinical Trial.
BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease with an annualised estimated glomerular filtration rate (eGFR) slope more negative than -2 mL/min/1.73 m(2)/year who had received agalsidase …
BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease wit …
Switch to agalsidase alfa after shortage of agalsidase beta in Fabry disease: a systematic review and meta-analysis of the literature.
Pisani A, Bruzzese D, Sabbatini M, Spinelli L, Imbriaco M, Riccio E. Pisani A, et al. Genet Med. 2017 Mar;19(3):275-282. doi: 10.1038/gim.2016.117. Epub 2016 Sep 8. Genet Med. 2017. PMID: 27608175 Free article.
BACKGROUND: In 2009, the agalsidase beta shortage resulted in switching to agalsidase alfa treatment for many Fabry disease patients, offering the unique opportunity to compare the effects of the two drugs. ...
BACKGROUND: In 2009, the agalsidase beta shortage resulted in switching to agalsidase alfa treatment for many Fabry disease pa …
Low-dose agalsidase beta treatment in male pediatric patients with Fabry disease: A 5-year randomized controlled trial.
Ramaswami U, Bichet DG, Clarke LA, Dostalova G, Fainboim A, Fellgiebel A, Forcelini CM, An Haack K, Hopkin RJ, Mauer M, Najafian B, Scott CR, Shankar SP, Thurberg BL, Tøndel C, Tylki-Szymanska A, Bénichou B, Wijburg FA. Ramaswami U, et al. Mol Genet Metab. 2019 May;127(1):86-94. doi: 10.1016/j.ymgme.2019.03.010. Epub 2019 Apr 3. Mol Genet Metab. 2019. PMID: 30987917 Free article. Clinical Trial.
Plasma GL-3 normalized and urine GL-3 reduced substantially. Higher anti-agalsidase beta antibody titers were associated with less robust SSCE GL-3 clearance and higher urine GL-3 levels. ...The current available evidence supports treatment of pediatric, classic mal …
Plasma GL-3 normalized and urine GL-3 reduced substantially. Higher anti-agalsidase beta antibody titers were associated with …
Historical Control Analysis Demonstrates Greater Long-Term Reduction in Plasma Globotriaosylceramide (Gb3) by Venglustat Compared With Placebo or Agalsidase Beta in Male Patients With Classic Fabry Disease.
Germain DP, DasMahapatra P, Liu S, Deegan P, Ortiz A, Modur V, Wilcox WR. Germain DP, et al. Mol Genet Genomic Med. 2025 Nov;13(11):e70152. doi: 10.1002/mgg3.70152. Mol Genet Genomic Med. 2025. PMID: 41246852 Free PMC article. Clinical Trial.
PURPOSE: To evaluate the disease biomarker response of venglustat in patients with Fabry disease (FD), utilizing data from a single-arm phase 2 study of venglustat and a placebo-controlled phase 3 study of agalsidase beta through historical control and case-matched …
PURPOSE: To evaluate the disease biomarker response of venglustat in patients with Fabry disease (FD), utilizing data from a single-arm phas …
Pharmacokinetics and pharmacodynamics of JR-051, a biosimilar of agalsidase beta, in healthy adults and patients with Fabry disease: Phase I and II/III clinical studies.
Nakamura K, Kawashima S, Tozawa H, Yamaoka M, Yamamoto T, Tanaka N, Yamamoto R, Okuyama T, Eto Y. Nakamura K, et al. Mol Genet Metab. 2020 Jul;130(3):215-224. doi: 10.1016/j.ymgme.2020.04.003. Epub 2020 May 1. Mol Genet Metab. 2020. PMID: 32389574 Free article. Clinical Trial.
Enzyme replacement therapy is currently the standard therapy for the disease, to which two alpha-galactosidase A formulations have been approved: agalsidase alpha (Replagal , Shire) and agalsidase beta (Fabrazyme , Sanofi). We have recently developed a biosimilar of …
Enzyme replacement therapy is currently the standard therapy for the disease, to which two alpha-galactosidase A formulations have been appr …
Enzyme replacement therapy for Anderson-Fabry disease.
El Dib R, Gomaa H, Carvalho RP, Camargo SE, Bazan R, Barretti P, Barreto FC. El Dib R, et al. Cochrane Database Syst Rev. 2016 Jul 25;7(7):CD006663. doi: 10.1002/14651858.CD006663.pub4. Cochrane Database Syst Rev. 2016. PMID: 27454104 Free PMC article.
No significant differences were found among the schedules for the primary efficacy outcome of self-assessed health state, or for pain scores.One trial comparing agalsidase alfa to agalsidase beta showed no significant difference for any adverse events such as dyspno …
No significant differences were found among the schedules for the primary efficacy outcome of self-assessed health state, or for pain scores …
First-in-human study with new recombinant agalsidase beta (ISU303) in healthy subjects.
Kim CO, Oh ES, Park MS. Kim CO, et al. J Clin Pharmacol. 2014 Jun;54(6):675-81. doi: 10.1002/jcph.262. Epub 2014 Mar 14. J Clin Pharmacol. 2014. PMID: 24408305 Clinical Trial.
ISU303 is a new recombinant agalsidase beta (Agal) enzyme replacement therapy under investigation for Fabry disease, caused by a deficiency in alpha-galactosidase A activity that leads to fatty deposits in tissues. ...
ISU303 is a new recombinant agalsidase beta (Agal) enzyme replacement therapy under investigation for Fabry disease, caused by …
Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease.
Germain DP, Waldek S, Banikazemi M, Bushinsky DA, Charrow J, Desnick RJ, Lee P, Loew T, Vedder AC, Abichandani R, Wilcox WR, Guffon N. Germain DP, et al. J Am Soc Nephrol. 2007 May;18(5):1547-57. doi: 10.1681/ASN.2006080816. Epub 2007 Apr 4. J Am Soc Nephrol. 2007. PMID: 17409312 Clinical Trial.
To determine the long-term safety and efficacy of recombinant human alpha-galactosidase A, an open-label, phase III extension study was conducted, involving 58 patients who had classic Fabry disease and completed a 20-wk, double-blind, randomized, placebo-controlled, phase III st …
To determine the long-term safety and efficacy of recombinant human alpha-galactosidase A, an open-label, phase III extension study was cond …
Enzyme replacement therapy for Anderson-Fabry disease.
El Dib RP, Nascimento P, Pastores GM. El Dib RP, et al. Cochrane Database Syst Rev. 2013 Feb 28;(2):CD006663. doi: 10.1002/14651858.CD006663.pub3. Cochrane Database Syst Rev. 2013. Update in: Cochrane Database Syst Rev. 2016 Jul 25;7:CD006663. doi: 10.1002/14651858.CD006663.pub4. PMID: 23450571 Updated.
Neither trial reported deaths.One of the three trials comparing agalsidase beta to placebo reported on globotriaosylceramide concentration in plasma and tissue and showed significant improvement: kidney, mean difference -1.70 (95% CI -2.09 to -1.31); heart, mean dif …
Neither trial reported deaths.One of the three trials comparing agalsidase beta to placebo reported on globotriaosylceramide c …
20 results