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

被引:0
作者
Olivier Morand
Jack Johnson
Jerry Walter
Leone Atkinson
Gregory Kline
Aline Frey
Juan Politei
Raphael Schiffmann
机构
[1] Azafaros B.V.,
[2] Fabry Support & Information Group,undefined
[3] National Fabry Disease Foundation,undefined
[4] Covance Inc.,undefined
[5] Idorsia Pharmaceuticals Ltd,undefined
[6] Laboratorio Neuroquímica Dr Chamoles (FESEN),undefined
[7] Baylor Scott & White Research Institute,undefined
来源
Advances in Therapy | 2019年 / 36卷
关键词
Clinical trial participation; Fabry disease; Fabry-specific pain; Gastrointestinal symptoms; Genetic disease; Patient survey; Quality of life; Treatment;
D O I
暂无
中图分类号
学科分类号
摘要
引用
收藏
页码:2866 / 2880
页数:14
相关论文
共 142 条
[1]  
Giugliani RN(2016)A 15-year perspective of the Fabry outcome survey J Inborn Errors Metab Screen. 4 1-12
[2]  
Niu D-M(2018)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 41 141-149
[3]  
Ramaswami U(2017)Characterization of classical and nonclassical Fabry disease: a multicenter study J Am Soc Nephrol 28 1631-1641
[4]  
West M(1999)Prevalence of lysosomal storage disorders JAMA 281 249-254
[5]  
Hughes D(2017)Newborn screening for lysosomal storage disorders in Illinois: the initial 15-month experience J Pediatr 190 130-135
[6]  
Kampmann C(2015)Lysosomal storage disorder screening implementation: findings from the first six months of full population pilot testing in Missouri J Pediatr 166 172-177
[7]  
Pintos-Morell G(2009)High incidence of the cardiac variant of Fabry disease revealed by newborn screening in the Taiwan Chinese population Circ Cardiovasc Genet. 2 450-456
[8]  
Arends M(2012)Neonatal screening for lysosomal storage disorders: feasibility and incidence from a nationwide study in Austria Lancet 379 335-341
[9]  
Korver S(2006)High incidence of later-onset fabry disease revealed by newborn screening Am J Hum Genet 79 31-40
[10]  
Hughes DA(2013)Newborn screening for Fabry disease in Japan: prevalence and genotypes of Fabry disease in a pilot study J Hum Genet 58 548-552