Symptoms and Quality of Life in Patients with Fabry Disease: Results from an International Patient Survey-Reference-Cited by-同舟云学术

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Symptoms and Quality of Life in Patients with Fabry Disease: Results from an International Patient Survey

Published:2019-08-21 Issue:10 Volume:36 Page:2866-2880
ISSN:0741-238X
Container-title:Advances in Therapy
language:en
Short-container-title:Adv Ther

Author:

Morand Olivier,Johnson Jack,Walter Jerry,Atkinson Leone,Kline Gregory,Frey Aline,Politei Juan,Schiffmann Raphael

Funder

Actelion Pharmaceuticals

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),General Medicine

Link

http://link.springer.com/content/pdf/10.1007/s12325-019-01061-x.pdf

Reference34 articles.

1. Giugliani RN, Niu D-M, Ramaswami U, West M, Hughes D, Kampmann C, Pintos-Morell G, et al. A 15-year perspective of the Fabry outcome survey. J Inborn Errors Metab Screen. 2016;4:1–12.

2. Arends M, Korver S, Hughes DA, Mehta A, Hollak CEM, Biegstraaten M. Phenotype, disease severity and pain are major determinants of quality of life in Fabry disease: results from a large multicenter cohort study. J Inherit Metab Dis. 2018;41(1):141–9.

3. Arends M, Wanner C, Hughes D, Mehta A, Oder D, Watkinson OT, et al. Characterization of classical and nonclassical Fabry disease: a multicenter study. J Am Soc Nephrol. 2017;28(5):1631–41.

4. Meikle PJ, Hopwood JJ, Clague AE, Carey WF. Prevalence of lysosomal storage disorders. JAMA. 1999;281(3):249–54.

5. Burton BK, Charrow J, Hoganson GE, Waggoner D, Tinkle B, Braddock SR, et al. Newborn screening for lysosomal storage disorders in Illinois: the initial 15-month experience. J Pediatr. 2017;190:130–5.

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1. Neuropathy and pain in Fabry disease;Rare Disease and Orphan Drugs Journal;2024-07-08

2. A systematic literature review on the health-related quality of life and economic burden of Fabry disease;Orphanet Journal of Rare Diseases;2024-04-30

3. Patient-reported experience with Fabry disease and its management in the real-world setting: results from a double-blind, cross-sectional survey of 280 respondents;Orphanet Journal of Rare Diseases;2024-04-11

4. FDrisk: development of a validated risk assessment tool for Fabry disease utilizing electronic health record data;Journal of Rare Diseases;2024-01-04

5. Fabry Disease in Women: Genetic Basis, Available Biomarkers, and Clinical Manifestations;Genes;2023-12-26

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