Cost-effectiveness of enzyme replacement therapy for Fabry disease
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[1] Wei Zheng,et al. ENZYME REPLACEMENT THERAPY FOR FABRY DISEASE , 2013 .
[2] W. Henley,et al. The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders. , 2012, Health technology assessment.
[3] S. Vegter,et al. Using a genetic, observational study as a strategy to estimate the potential cost-effectiveness of pharmacological CCR5 blockade in dialysis patients , 2011, Pharmacogenetics and genomics.
[4] S. Simoens. Pricing and reimbursement of orphan drugs: the need for more transparency , 2011, Orphanet journal of rare diseases.
[5] T. Cox,et al. Orphan drug pricing may warrant a competition law investigation , 2010, BMJ : British Medical Journal.
[6] D. Bichet,et al. Baseline characteristics of patients enrolled in the Canadian Fabry Disease Initiative. , 2010, Molecular genetics and metabolism.
[7] Y. Chien,et al. Newborn screening for Fabry disease in Taiwan reveals a high incidence of the later‐onset GLA mutation c.936+919G>A (IVS4+919G>A) , 2009, Human mutation.
[8] A. Garg,et al. Angiotensin inhibition in renovascular disease: a population-based cohort study. , 2008, American heart journal.
[9] R. Brady,et al. Elevated globotriaosylsphingosine is a hallmark of Fabry disease , 2008, Proceedings of the National Academy of Sciences.
[10] G. Sommer,et al. Reference , 2008 .
[11] J. M. Aerts,et al. Treatment of Fabry Disease: Outcome of a Comparative Trial with Agalsidase Alfa or Beta at a Dose of 0.2 mg/kg , 2007, PloS one.
[12] J. Charrow,et al. Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease. , 2007, Journal of the American Society of Nephrology : JASN.
[13] F. Bemelman,et al. The Dutch Fabry cohort: Diversity of clinical manifestations and Gb3 levels , 2007, Journal of Inherited Metabolic Disease.
[14] R. Desnick,et al. High incidence of later-onset fabry disease revealed by newborn screening. , 2006, American journal of human genetics.
[15] M. Connock,et al. A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1. , 2006, Health technology assessment.
[16] D. Hughes,et al. Drugs for exceptionally rare diseases: do they deserve special status for funding? , 2005, QJM : monthly journal of the Association of Physicians.
[17] M. Postma,et al. Need for differential discounting of costs and health effects in cost effectiveness analyses , 2005, BMJ : British Medical Journal.
[18] Paul F. M. Krabbe,et al. Kwaliteit van leven meten in economische evaluaties: het Nederlands EQ-5D-tarief , 2005 .
[19] A. Mehta,et al. Effects of enzyme replacement therapy on pain and health related quality of life in patients with Fabry disease: data from FOS (Fabry Outcome Survey) , 2005, Journal of Medical Genetics.
[20] J. Kriegsmann,et al. Anderson–Fabry disease: Clinical manifestations of disease in female heterozygotes , 2001, Journal of Inherited Metabolic Disease.
[21] K. Macdermot,et al. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males , 2001, Journal of medical genetics.
[22] K. Macdermot,et al. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females , 2001, Journal of medical genetics.
[23] S. Yusuf,et al. Effects of Ramipril on Coronary Events in High-Risk Persons: Results of the Heart Outcomes Prevention Evaluation Study , 2001, Circulation.
[24] S. Yusuf,et al. Effects of ramipril on cardiovascular and microvascular outcomes in people with diabetes mellitus: results of the HOPE study and MICRO-HOPE substudy. Heart Outcomes Prevention Evaluation Study Investigators. , 2000 .
[25] S. Yusuf,et al. Effects of an angiotensin-converting-enzyme inhibitor, ramipril, on cardiovascular events in high-risk patients. The Heart Outcomes Prevention Evaluation Study Investigators. , 2000 .
[26] Andrew Briggs,et al. An Introduction to Markov Modelling for Economic Evaluation , 1998, PharmacoEconomics.
[27] P. Dolan,et al. Modeling valuations for EuroQol health states. , 1997, Medical care.
[28] S. Tsuji. [Alpha-galactosidase A deficiency--Fabry's disease]. , 1988, Tanpakushitsu kakusan koso. Protein, nucleic acid, enzyme.
[29] J. Brecht,et al. Cost Effectiveness of Ramipril in Patients at High Risk for Cardiovascular Events , 2012, PharmacoEconomics.
[30] M. Pitt,et al. Dasatinib and nilotinib for imatinib-resistant or -intolerant chronic myeloid leukaemia: a systematic review and economic evaluation. , 2012, Health technology assessment.
[31] R. Schiffmann,et al. Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry-Anderson disease. , 2007, PharmacoEconomics.
[32] E. C. Ham. Body composition and exercise intolerance in renal transplant patients: the response to exercise training , 2006 .
[33] S. Packman,et al. Agalsidase-Beta Therapy for Advanced Fabry Disease A Randomized Trial , 2006 .
[34] P. Stalmeier,et al. [Measuring the quality of life in economic evaluations: the Dutch EQ-5D tariff]. , 2005, Nederlands tijdschrift voor geneeskunde.
[35] D. Blom,et al. Recombinant Enzyme Therapy for Fabry Disease: Absence of Editing of Human α-Galactosidase A mRNA , 2003 .
[36] D. Blom,et al. UvA-DARE ( Digital Academic Repository ) Recombinant enzyme therapy for Fabry disease : absence of editing of human alpha-galactosidase A mRNA , 2002 .
[37] Z. Stark,et al. Orphanet Journal of Rare Diseases , 2022 .
[38] S. Simoens. Clinicoeconomics and Outcomes Research Dovepress Biosimilar Medicines and Cost-effectiveness , 2022 .