Results 1 to 10 of about 1,001 (98)

New insights in efficacy of different ERT dosages in Fabry disease: Switch and switch-back studies data following agalsidase beta shortage. Update of systematic review [PDF]

Genetics in Medicine Open, 2023
In 2016, a systematic review and a meta-analysis of existing data on the effects of switch from agalsidase beta to alfa in patients with Fabry disease showed that the switch was well tolerated and associated with stable disease progression.
Massimo Imbriaco, Pasquale Buonanno, Carlo Garofalo   +2 more
exaly   +4 more sources

Stepwise shortening of agalsidase beta infusion duration in Fabry disease: Clinical experience with infusion rate escalation protocol [PDF]

Molecular Genetics & Genomic Medicine, 2021
Background Although enzyme replacement therapy with agalsidase beta resulted in a variety of clinical benefits, life‐long biweekly intravenous infusion may impact on patients’ quality of life.
Pasquale Buonanno, Eleonora Riccio, Monica Franzese   +2 more
exaly   +3 more sources

A systematic literature review to evaluate the cardiac and cerebrovascular outcomes of patients with Fabry disease treated with agalsidase Beta [PDF]

Frontiers in Cardiovascular Medicine
BackgroundAgalsidase beta is used to treat Fabry disease (FD); however, data on cardiac and cerebrovascular outcomes with agalsidase beta treatment come from studies with limited numbers of patients.MethodsA systematic literature review of studies ...
Pronabesh DasMahapatra
exaly   +4 more sources

Development of a Biosimilar of Agalsidase Beta for the Treatment of Fabry Disease: Preclinical Evaluation [PDF]

Drugs in R&D, 2023
Background and Objective Fabry disease (FD) is a rare lysosomal storage disorder caused by a deficiency of the enzyme α-galactosidase A (aGal A). Since 2001, two different enzyme replacement therapies have been authorized, with agalsidase beta being used
André B. P. van Kuilenburg, Carla E. M. Hollak, Ana Travella, Melisa Jacobs, Lucas D. Gentilini, René Leen, Karen M. M. Ghauharali-van der Vlugt, Femke S. Beers Stet, Susan M. I. Goorden, Sanne van der Veen, Marcelo Criscuolo, Mariana Papouchado   +11 more
doaj   +2 more sources

Safety and Tolerability of a Shorter Agalsidase Beta Infusion Time in Patients with Classic or Later-Onset Fabry Disease [PDF]

Biomedicines
Background: The multisystem manifestations of Fabry disease can create major challenges in patient care. Although enzyme replacement therapy with recombinant agalsidase beta has demonstrated clinical benefits, the standard fortnightly, multi-hour ...
Dominique P. Germain, Alice Porto Vasconcelos, Lien Tran Thi Phuong, Najya Bedreddine, Mihaela Turcan, Wenting Trang, Lynda Barache   +6 more
doaj   +2 more sources

Clinical outcomes of agalsidase Beta (fabrazyme) in Chinese fabry disease patients with proteinuria: a case series [PDF]

Frontiers in Pediatrics
BackgroundFabry disease (FD) is a rare X-linked lysosomal storage disorder caused by pathogenic GLA gene mutations, leading to deficient α-galactosidase A (α-GalA) activity and systemic accumulation of globotriaosylceramide (Gb3) and its derivatives.
Zelei He, Yuanyuan Wu, Hongmei Yang, Yijuan Li, Lingling Xu   +4 more
doaj   +2 more sources

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 [PDF]

Molecular Genetics & Genomic Medicine
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 ...
Dominique P. Germain, Pronabesh DasMahapatra, Shiguang Liu, Patrick Deegan, Alberto Ortiz, Vijay Modur, William R. Wilcox   +6 more
doaj   +2 more sources

Comparative pharmacokinetics and pharmacodynamics of two formulations of agalsidase beta (agalsidase Biosidus) and Fabrazyme® by intravenous infusion in healthy male volunteers [PDF]

Molecular Genetics and Metabolism Reports
Fabry disease is a rare X-linked lysosomal condition that leads to the accumulation of glycosphingolipids in various tissues, causing cellular dysfunction, tissue remodeling, progressive fibrosis, and organ failure.
Viridiana Berstein, Eduardo M. Pirotzky, Hernán D. Taconelli, M. Gabriela Gobbi, Lara Beider, Natali D. Salgueiro, Laila Dome, Roberto A. Diez, Hugo Sotelo, Sabrina Coppola   +9 more
doaj   +2 more sources

Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis [PDF]

ESC Heart Failure, 2020
Aims Long‐term treatment effect studies in large female Fabry patient groups are challenging to design because of phenotype heterogeneity and lack of appropriate comparison groups, and have not been reported.
Christoph Wanner, Ulla Feldt‐Rasmussen, Ana Jovanovic, Aleš Linhart, Meng Yang, Elvira Ponce, Eva Brand, Dominique P. Germain, Derralynn A. Hughes, John L. Jefferies, Ana Maria Martins, Albina Nowak, Bojan Vujkovac, Frank Weidemann, Michael L. West, Alberto Ortiz   +15 more
doaj   +2 more sources

A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease [PDF]

Orphanet Journal of Rare Diseases
Background This is the first phase 4 study evaluating safety and efficacy of enzyme replacement therapy (ERT) in Chinese patients with Fabry disease, and exploring the impact of COVID-19 infection on the prognosis of Fabry disease under ERT.
Hong Ren, Wei Zhang, Yan Ouyang, Junhong Guo, Hong Xu, Jie Ma, Xiaoping Luo, Xiaoxia Pan, Yun Yuan, Wei Zhang, Qian Shen, Bin Li, Qiqi Feng, Shi Liu, Nan Chen   +14 more
doaj   +2 more sources