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Fabrazyme

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https://www.readbyqxmd.com/read/27902397/vesivirus-2117-capsids-more-closely-resemble-sapovirus-and-lagovirus-particles-than-other-known-vesivirus-structures
#1
Michaela Conley, Edward Emmott, Richard Orton, David Taylor, Daniel Carneiro, Kazuyoshi Murata, Ian Goodfellow, Grant Hansman, David Bhella
Vesivirus 2117 is an adventitious agent that in 2009, was identified as a contaminant of CHO cells propagated in bioreactors at a pharmaceutical manufacturing plant belonging to Genzyme. The consequent interruption in supply of Fabrazyme and Cerezyme (drugs used to treat Fabry and Gaucher disease respectively), caused significant economic losses. Vesivirus 2117 is a member of the Caliciviridae; a family of small icosahedral viruses encoding a positive sense RNA genome. We have used cryo-electron microscopy and three dimensional image reconstruction to calculate a structure of vesivirus 2117 virus like particles as well as feline calicivirus and a chimeric sapovirus...
November 14, 2016: Journal of General Virology
https://www.readbyqxmd.com/read/26252393/oral-migalastat-hcl-leads-to-greater-systemic-exposure-and-tissue-levels-of-active-%C3%AE-galactosidase-a-in-fabry-patients-when-co-administered-with-infused-agalsidase
#2
David G Warnock, Daniel G Bichet, Myrl Holida, Ozlem Goker-Alpan, Kathy Nicholls, Mark Thomas, Francois Eyskens, Suma Shankar, Mathews Adera, Sheela Sitaraman, Richie Khanna, John J Flanagan, Brandon A Wustman, Jay Barth, Carrolee Barlow, Kenneth J Valenzano, David J Lockhart, Pol Boudes, Franklin K Johnson
UNLABELLED: Migalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological chaperone for the treatment of α-galactosidase A (α-Gal A) deficiency, which leads to Fabry disease, an X-linked, lysosomal storage disorder. The currently approved, biologics-based therapy for Fabry disease is enzyme replacement therapy (ERT) with either agalsidase alfa (Replagal) or agalsidase beta (Fabrazyme). Based on preclinical data, migalastat HCl in combination with agalsidase is expected to result in the pharmacokinetic (PK) enhancement of agalsidase in plasma by increasing the systemic exposure of active agalsidase, thereby leading to increased cellular levels in disease-relevant tissues...
2015: PloS One
https://www.readbyqxmd.com/read/25098458/-the-switch-of-enzyme-therapy-in-fabry-disease
#3
REVIEW
Eleonora Riccio, Antonio Pisani
Fabry disease (FD) is a multiorgan X-linked lysosomal storage disorder resulted from the deficiency of the lysosomal enzyme alpha galactosidase A. It particularly affects the heart, kidneys and cerebrovascular system. The treatment options for FD patients include long-term enzyme replacement therapy (ERT). Two recombinant enzyme formulations for the ERT of FD are available on European market: agalsidase alfa and agalsidase beta. Numerous evidences in the literature have confirmed the safety and efficacy of ERT...
July 2014: Giornale Italiano di Nefrologia: Organo Ufficiale Della Società Italiana di Nefrologia
https://www.readbyqxmd.com/read/24886109/fabry-nephropathy-a-review-how-can-we-optimize-the-management-of-fabry-nephropathy
#4
REVIEW
Stephen Waldek, Sandro Feriozzi
Fabry disease is a rare, X-linked, lysosomal storage disease caused by mutations in the gene encoding the enzyme alpha-galactosidase A. Complete or partial deficiency in this enzyme leads to intracellular accumulation of globotriaosylceramide (Gb3) and related glycosphingolipids in many cell types throughout the body, including the kidney. Progressive accumulation of Gb3 in podocytes, epithelial cells and the tubular cells of the distal tubule and loop of Henle contribute to the renal symptoms of Fabry disease, which manifest as proteinuria and reduced glomerular filtration rate leading to chronic kidney disease and progression to end-stage renal disease...
May 6, 2014: BMC Nephrology
https://www.readbyqxmd.com/read/24718841/successful-management-of-enzyme-replacement-therapy-in-related-fabry-disease-patients-with-severe-adverse-events-by-switching-from-agalsidase-beta-fabrazyme-%C3%A2-to-agalsidase-alfa-replagal-%C3%A2
#5
Kazuya Tsuboi, Hiroshi Yamamoto, Fuji Somura, Hiromi Goto
BACKGROUND: Enzyme replacement therapy (ERT) is the only approved therapy for Fabry disease. In June 2009, there was a worldwide shortage of agalsidase beta, necessitating dose reductions or switching to agalsidase alfa in some patients. CASE PRESENTATION: We present two cases of Fabry disease (a parent and a child) who received agalsidase beta for 27 months at the licensed dose and 10 months at a reduced dose, followed by a switch to agalsidase alfa for 28 months...
2015: JIMD Reports
https://www.readbyqxmd.com/read/24449601/single-step-affinity-purification-of-enzyme-biotherapeutics-a-platform-methodology-for-accelerated-process-development
#6
Kevin P Brower, Venkat K Ryakala, Ryan Bird, Rahul Godawat, Frank J Riske, Konstantin Konstantinov, Veena Warikoo, Jean Gamble
Downstream sample purification for quality attribute analysis is a significant bottleneck in process development for non-antibody biologics. Multi-step chromatography process train purifications are typically required prior to many critical analytical tests. This prerequisite leads to limited throughput, long lead times to obtain purified product, and significant resource requirements. In this work, immunoaffinity purification technology has been leveraged to achieve single-step affinity purification of two different enzyme biotherapeutics (Fabrazyme® [agalsidase beta] and Enzyme 2) with polyclonal and monoclonal antibodies, respectively, as ligands...
May 2014: Biotechnology Progress
https://www.readbyqxmd.com/read/24402625/-enzyme-replacement-therapy-in-patients-with-fabry-disease-state-of-the-art-and-review-of-the-literature
#7
REVIEW
Eleonora Riccio, Ivana Capuano, Bianca Visciano, Cristina Marchetiello, Fortunato Petrillo, Antonio Pisani
Anderson-Fabry disease is an X-linked lysosomal storage disorder resulting from the deficiency of the hydrolytic enzyme alpha galactosidase A, with consequent accumulation of globotrioasoyl ceramide in cells and tissues of the body, resulting in a multi-system pathology including end organ failure. In the classical phenotype, cardiac failure, renal failure and stroke result and a reduced median life expectancy ensues. The current causal treatment for Fabry disease is enzyme replacement therapy (ERT). Two different products, Replagal (agalsidase alfa) and Fabrazyme (agalsidase beta), have been commercially available in Europe for almost 10 years and they are both indicated for long-term treatment...
September 2013: Giornale Italiano di Nefrologia: Organo Ufficiale Della Società Italiana di Nefrologia
https://www.readbyqxmd.com/read/24388678/clinical-observations-on-enzyme-replacement-therapy-in-patients-with-fabry-disease-and-the-switch-from-agalsidase-beta-to-agalsidase-alfa
#8
Hsiang-Yu Lin, Yu-Hsiu Huang, Hsuan-Chieh Liao, Hao-Chuan Liu, Ting-Rong Hsu, Chia-I Shen, Shao-Tzu Li, Cheng-Fang Li, Li-Hong Lee, Pi-Chang Lee, Chun-Kai Huang, Chuan-Chi Chiang, Shuan-Pei Lin, Dau-Ming Niu
BACKGROUND: Fabry disease is an X-linked inherited lysosomal storage disease that can be treated with the enzymes of agalsidase beta (Fabrazyme) and agalsidase alfa (Replagal). Since June 2009, viral contamination of Genzyme's production facility has resulted in a worldwide shortage of agalsidase beta, leading to the switch to agalsidase alfa for patients with Fabry disease in Taiwan. METHODS: The medical records were retrospectively reviewed for nine male patients with Fabry disease from the start of agalsidase beta treatment until the switch to agalsidase alfa for at least 1 year...
April 2014: Journal of the Chinese Medical Association: JCMA
https://www.readbyqxmd.com/read/23832445/-fabry-s-disease-a-comparison-of-the-effectiveness-of-two-different-enzyme-replacement-therapies
#9
COMPARATIVE STUDY
Antonio Pisani, Bianca Visciano, Ivana Capuano, Antonello Mancini, Eleonora Riccio
INTRODUCTION: Anderson-Fabry disease (AFD) is a multiorgan X-linked lysosomal storage disease that particularly affects the heart, kidneys, and cerebrovascular system. Current treatment is with enzyme replacement therapy (ERT), using either beta-galactosidase ('Fabrazyme') or alpha-galactosidase ('Replagal'). From June 2009, it was recommended that patients switch to alpha-galactosidase due to a manufacturing shortage of beta-galactosidase. This study assesses the effect of switching to alpha-galactosidase on clinical outcomes in patients with AFD previously treated with beta-galactosidase...
January 2013: Giornale Italiano di Nefrologia: Organo Ufficiale Della Società Italiana di Nefrologia
https://www.readbyqxmd.com/read/23430871/effect-of-reduced-agalsidase-beta-dosage-in-fabry-patients-the-australian-experience
#10
Joanna Ghali, Kathy Nicholls, Charles Denaro, David Sillence, Ian Chapman, Jack Goldblatt, Mark Thomas, Janice Fletcher
BACKGROUND: In Australia, enzyme replacement therapy (ERT) for Fabry Disease (FD), both Agalsidase alfa (Replagal, Shire HGT) and beta (Fabrazyme, Genzyme), is funded and monitored through a specific government program. Agalsidase beta supply has been rationed by Genzyme since 2009 due to manufacturing issues. Consequently, the Australian Fabry Disease Advisory Committee has treated patients on Agalsidase beta at 50% of their usual dose from mid-2009, with a further reduction to 30% for some patients from late 2009...
2012: JIMD Reports
https://www.readbyqxmd.com/read/23430546/effects-of-switching-from-agalsidase-beta-to-agalsidase-alfa-in-10-patients-with-anderson-fabry-disease
#11
A Pisani, L Spinelli, B Visciano, I Capuano, M Sabbatini, E Riccio, G Messalli, M Imbriaco
Anderson-Fabry disease (AFD) is a multiorgan X-linked lysosomal storage disease that particularly affects the heart, kidneys, and cerebrovascular system. Current treatment is enzyme replacement therapy (ERT) with agalsidase beta (Fabrazyme(®), Genzyme Corporation, Cambridge, MA, USA) or agalsidase alfa (Replagal(®), Shire Human Genetic Therapies AB, Lund, Sweden). It was recommended that patients switch to agalsidase alfa due to a manufacturing shortage of agalsidase beta beginning in June 2009. This study assessed the effect of switching to agalsidase alfa on clinical outcomes in patients with AFD previously treated with agalsidase beta...
2013: JIMD Reports
https://www.readbyqxmd.com/read/23089251/the-effectiveness-and-cost-effectiveness-of-enzyme-and-substrate-replacement-therapies-a-longitudinal-cohort-study-of-people-with-lysosomal-storage-disorders
#12
MULTICENTER STUDY
K Wyatt, W Henley, L Anderson, R Anderson, V Nikolaou, K Stein, L Klinger, D Hughes, S Waldek, R Lachmann, A Mehta, A Vellodi, S Logan
OBJECTIVES: To determine natural history and estimate effectiveness and cost of enzyme replacement therapy (ERT) and substrate replacement therapy (SRT) for patients with Gaucher disease, Fabry disease, mucopolysaccharidosis type I (MPS I), mucopolysaccharidosis type II (MPS II), Pompe disease and Niemann-Pick type C (NPC) disease. DESIGN: Cohort study including prospective and retrospective clinical- and patient-reported data. Age- and gender-adjusted treatment effects were estimated using generalised linear mixed models...
2012: Health Technology Assessment: HTA
https://www.readbyqxmd.com/read/22963910/enzyme-replacement-therapy-in-patients-with-fabry-disease-state-of-the-art-and-review-of-the-literature
#13
REVIEW
Antonio Pisani, Bianca Visciano, Graciana Diez Roux, Massimo Sabbatini, Caterina Porto, Giancarlo Parenti, Massimo Imbriaco
Anderson-Fabry disease is an X-linked lysosomal storage disorder resulting from the deficiency of the hydrolytic enzyme alpha galactosidase A, with consequent accumulation of globotrioasoyl ceramide in cells and tissues of the body, resulting in a multi-system pathology including end organ failure. In the classical phenotype, cardiac failure, renal failure and stroke result in a reduced median life expectancy. The current causal treatment for Fabry disease is the enzyme replacement therapy (ERT): two different products, Replagal (agalsidase alfa) and Fabrazyme (agalsidase beta), have been commercially available in Europe for almost 10 years and they are both indicated for long-term treatment...
November 2012: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/22878505/clinical-observation-of-patients-with-fabry-disease-after-switching-from-agalsidase-beta-fabrazyme-to-agalsidase-alfa-replagal
#14
Kazuya Tsuboi, Hiroshi Yamamoto
PURPOSE: Fabry disease is a rare, X-linked, inherited lysosomal storage disorder that can be treated with the enzymes agalsidasealfa (Replagal) and agalsidase beta (Fabrazyme). Currently, there is a global shortage of agalsidase beta, and this has increased global demand for agalsidase alfa. We assess the feasibility of switching patients on agalsidase beta treatment to agalsidase alfa instead. METHODS: This analysis is part of an ongoing observational study involving 11 patients with Fabry disease in whom the treatment was switched from agalsidase beta (1 mg/kg every other week) to agalsidase alfa (0...
September 2012: Genetics in Medicine: Official Journal of the American College of Medical Genetics
https://www.readbyqxmd.com/read/22498845/clinical-observation-of-patients-with-fabry-disease-after-switching-from-agalsidase-beta-fabrazyme-to-agalsidase-alfa-replagal
#15
Kazuya Tsuboi, Hiroshi Yamamoto
PURPOSE: Fabry disease is a rare, X-linked, inherited lysosomal storage disorder that can be treated with the enzymes agalsidase alfa (Replagal) and agalsidase beta (Fabrazyme). Currently, there is a global shortage of agalsidase beta, and this has increased global demand for agalsidase alfa. We assess the feasibility of switching patients on agalsidase beta treatment to agalsidase alfa instead. METHODS: This analysis is part of an ongoing observational study involving 11 patients with Fabry disease in whom the treatment was switched from agalsidase beta (1 mg/kg every other week) to agalsidase alfa (0...
September 2012: Genetics in Medicine: Official Journal of the American College of Medical Genetics
https://www.readbyqxmd.com/read/21755431/progressive-renal-failure-despite-long-term-biweekly-enzyme-replacement-therapy-in-a-patient-with-fabry-disease-secondary-to-a-new-%C3%AE-galactosidase-mutation-of-leu311arg-l311r
#16
REVIEW
Keisuke Suzuki, Naoto Miura, Wataru Kitagawa, Shinkichi Suzuki, Atsushi Komatsuda, Kazuhiro Nishikawa, Daisuke Watanabe, Hirokazu Imai
A 37-year-old Japanese man affected by Fabry disease secondary to a novel mutation of Leu311Arg (L311R) in α-galactosidase demonstrated progressive renal failure despite biweekly enzyme replacement therapy (ERT) for approximately 10 years. Kidney biopsy revealed foamy glomerular epithelial cells, compatible with the typical pathologic features of Fabry disease. The patient entered a phase III study of Replagal (agalsidase alfa) in 2001, allowing him to continue ERT with biweekly dosing for almost 10 years...
December 2011: Clinical and Experimental Nephrology
https://www.readbyqxmd.com/read/21416197/biotherapeutic-target-or-sink-analysis-of-the-macrophage-mannose-receptor-tissue-distribution-in-murine-models-of-lysosomal-storage-diseases
#17
Xin Sheen Zhang, William Brondyk, John T Lydon, Beth L Thurberg, Peter A Piepenhagen
Lysosomal storage diseases (LSDs) are metabolic disorders caused by enzyme deficiencies that lead to lysosomal accumulation of undegraded substrates. Enzyme replacement therapies (ERT) have been developed as treatments for patients with Gaucher, Niemann-Pick, Fabry, and Pompe diseases. Depending on the disease, the corresponding therapeutic enzyme is designed to be internalized by diseased cells through receptor-mediated endocytosis via macrophage mannose receptors (MMR) or mannose-6-phosphate receptors (M6PR)...
June 2011: Journal of Inherited Metabolic Disease
https://www.readbyqxmd.com/read/21299550/modelling-the-resource-implications-of-managing-adults-with-fabry-disease-in-italy
#18
Julian F Guest, Daniela Concolino, Raffaele Di Vito, Claudio Feliciani, Rossella Parini, Anna Zampetti
AIMS: This study estimated the resource implications and budget impact of managing adults with Fabry disease in Italy, from the perspective of the Servizio Sanitario Nazionale (SSN). METHODS: A decision model was constructed using published clinical outcomes and clinician-derived resource utilisation estimates depicting the management of adults with Fabry disease in Italy. RESULTS: The expected annual cost of managing 220 existing and 20 new Fabry patients in Italy was estimated to be €28·3 million...
July 2011: European Journal of Clinical Investigation
https://www.readbyqxmd.com/read/21211680/-enzyme-replacement-therapy-of-lysosomal-storage-diseases
#19
D P Germain, C Boucly, R Y Carlier, E Caudron, P Charlier, F Colas, F Jabbour, V Martinez, S Mokhtari, D Orlikowski, N Pellegrini, C Perronne, H Prigent, R Rubinsztajn, K Benistan
Extraction and purification of an acid β-glucosidase from human placenta (alglucerase) for the treatment of Gaucher disease, replaced a few years later by a recombinant enzyme (imiglucérase, Cerezyme(®)), has paved the way to the development of enzyme replacement therapies (ERT) for the treatment of lysosomal storage diseases (LSD) among which Fabry disease for which the long-term efficacy of the two currently available preparations (agalsidase alfa, Replagal(®) and Fabrazyme(®)) is still being investigated...
December 2010: La Revue de Médecine Interne
https://www.readbyqxmd.com/read/21111645/the-fabrazyme-shortage-a-call-to-action-for-metabolic-physicians
#20
COMMENT
Sandra Sirrs
The recent shortages of enzyme replacement therapy for Fabry disease have highlighted areas of vulnerability for patients who require this treatment. Guidelines on allocation of limited stock of enzyme replacement therapy are of use for clinicians dealing with the current shortages. However, the community of metabolic physicians must advocate for changes that will minimize the impact of future drug shortages for their patients with lysosomal storage diseases.
January 2011: Molecular Genetics and Metabolism
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