scholarly journals Recommendations on Reintroduction of Agalsidase Beta for Patients with Fabry Disease in Europe, Following a Period of Shortage

2012 ◽  
pp. 51-56 ◽  
Author(s):  
Gabor E. Linthorst ◽  
Alessandro P. Burlina ◽  
Franco Cecchi ◽  
Timothy M. Cox ◽  
Janice M. Fletcher ◽  
...  
Keyword(s):  
Fabry Disease ◽  
Agalsidase Beta

Dual therapy with migalastat and agalsidase-beta in a patient with Fabry disease with progressing hypertrophic cardiomyopathy

2021 ◽  
Vol 132 (2) ◽  
pp. S103
Author(s):  
Chanan Stauffer ◽  
Manisha Balwani ◽  
Robert J. Desnick ◽  
Luca Fierro ◽  
Jaya Ganesh
Keyword(s):  
Hypertrophic Cardiomyopathy ◽  
Fabry Disease ◽  
Dual Therapy ◽  
Agalsidase Beta

scholarly journals Agalsidase alfa and agalsidase beta in the treatment of Fabry disease: does the dose really matter?

2014 ◽  
Vol 17 (1) ◽  
pp. 21-23 ◽  
Author(s):  
Antonio Pisani ◽  
Eleonora Riccio ◽  
Massimo Sabbatini
Keyword(s):  
Fabry Disease ◽  
Agalsidase Beta ◽  
Agalsidase Alfa

Female Fabry disease: significant improvement of Fabry disease-related gastrointestinal symptoms in a large cohort of female patients treated with agalsidase beta: data from the Fabry Registry

2014 ◽  
Vol 111 (2) ◽  
pp. S114-S115
Author(s):  
William Wilcox ◽  
Ulla Feldt-Rasmussen ◽  
Ana Maria Martins ◽  
Alberto Ortiz ◽  
Frank Weidemann ◽  
...  
Keyword(s):  
Fabry Disease ◽  
Gastrointestinal Symptoms ◽  
Large Cohort ◽  
Agalsidase Beta ◽  
Female Patients

scholarly journals Improvement of Fabry Disease-Related Gastrointestinal Symptoms in a Significant Proportion of Female Patients Treated with Agalsidase Beta: Data from the Fabry Registry

2017 ◽  
pp. 45-51 ◽  
Author(s):  
William R. Wilcox ◽  
Ulla Feldt-Rasmussen ◽  
Ana Maria Martins ◽  
Alberto Ortiz ◽  
Roberta M. Lemay ◽  
...  
Keyword(s):  
Fabry Disease ◽  
Significant Proportion ◽  
Gastrointestinal Symptoms ◽  
Agalsidase Beta ◽  
Female Patients

Efficacy and safety of JR-051, a biosimilar of agalsidase beta, in patients with Fabry disease; results of a multicenter, open-label phase 3 study in Japan

2018 ◽  
Vol 123 (2) ◽  
pp. S79
Author(s):  
Minako Kobayashi ◽  
Tatsuyoshi Yamamoto ◽  
Mariko Yamaoka ◽  
Teiji Tomio
Keyword(s):  
Fabry Disease ◽  
Agalsidase Beta ◽  
Efficacy And Safety ◽  
Open Label ◽  
Phase 3 ◽  
Open Label Phase

Treatment switch in Fabry disease- a matter of dose?

2020 ◽  
pp. jmedgenet-2020-106874
Author(s):  
Malte Lenders ◽  
Peter Nordbeck ◽  
Sima Canaan-Kühl ◽  
Lukas Kreul ◽  
Thomas Duning ◽  
...  
Keyword(s):  
Fabry Disease ◽  
Dose Group ◽  
Clinical Symptoms ◽  
Organ Damage ◽  
Agalsidase Beta ◽  
Disease Epidemiology ◽  
Reduced Dose ◽  
Clinical Events ◽  
Agalsidase Alfa ◽  
Switch Group

BackgroundPatients with Fabry disease (FD) on reduced dose of agalsidase-beta or after switch to agalsidase-alfa show a decline in chronic kidney disease epidemiology collaboration-based estimated glomerular filtration rate (eGFR) and a worsened plasma lyso-Gb3 decrease. Hence, the most effective dose is still a matter of debate.MethodsIn this prospective observational study, we assessed end-organ damage and clinical symptoms in 78 patients who had received agalsidase-beta (1.0 mg/kg) for >1 year, which were assigned to continue this treatment (agalsidase-beta, regular-dose group, n=17); received a reduced dose of agalsidase-beta and subsequent switch to agalsidase-alfa (0.2 mg/kg) or a direct switch to 0.2 mg/kg agalsidase-alfa (switch group, n=22); or were re-switched to agalsidase-beta after receiving agalsidase-alfa for 12 months (re-switch group, n=39) with a follow-up of 88±25 months.ResultsNo differences for clinical events were observed for all groups. Patients within the re-switch group started with the worst eGFR values at baseline (p=0.0217). Overall, eGFR values remained stable in the regular-dose group (p=0.1052) and decreased significantly in the re-switch and switch groups (p<0.0001 and p=0.0052, respectively). However, in all groups males presented with an annual loss of eGFR by –2.9, –2.5 and −3.9 mL/min/1.73 m² (regular-dose, re-switch, switch groups, all p<0.05). In females, eGFR decreased significantly only in the re-switch group by −2.9 mL/min/1.73 m² per year (p<0.01). Lyso-Gb3 decreased in the re-switch group after a change back to agalsidase-beta (p<0.05).ConclusionsOur data suggest that a re-switch to high dosage of agalsidase results in a better biochemical response, but not in a significant renal amelioration especially in classical males.

Burden of Fabry disease in young patients (≤30 years of age) who were initiated on enzyme replacement therapy with agalsidase beta: a Fabry Registry analysis

2017 ◽  
Vol 120 (1-2) ◽  
pp. S65-S66
Author(s):  
Robert J. Hopkin ◽  
Eva Brand ◽  
Ulla F. Rasmussen ◽  
Dominique P. Germain ◽  
Nathalie Guffon ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Fabry Disease ◽  
Replacement Therapy ◽  
Young Patients ◽  
Agalsidase Beta ◽  
Enzyme Replacement ◽  
Registry Analysis

Enzyme replacement therapy in Fabry disease: Comparison of agalsidase alfa and agalsidase beta

2008 ◽  
Vol 95 (1-2) ◽  
pp. 114-115 ◽  
Author(s):  
Atul Mehta ◽  
Michael Beck ◽  
Christoph Kampmann ◽  
Andrea Frustaci ◽  
Dominique P. Germain ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Fabry Disease ◽  
Replacement Therapy ◽  
Agalsidase Beta ◽  
Enzyme Replacement ◽  
Agalsidase Alfa
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