Adherence to a pharmacological chaperone therapy among patients with Fabry disease: One centre experience

被引:0
|
作者
Gorton, Janet [1 ]
Forshaw-Hulme, Stuart [1 ]
Jones, Mairead [1 ]
Jovanovic, Ana [1 ]
Stepien, Karolina [1 ]
机构
[1] Salford Royal NHS Fdn Trust, Manchester, Lancs, England
关键词
D O I
10.1016/j.ymgme.2018.12.148
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
132
引用
收藏
页码:S63 / S63
页数:1
相关论文
共 50 条
  • [1] Pharmacological chaperone therapy for Fabry disease
    Ishii, Satoshi
    [J]. PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES, 2012, 88 (01): : 18 - 30
  • [2] A pharmacogenetic approach to pharmacological chaperone therapy for Fabry disease
    Benjamin, Elfrida
    Wu, Xiaoyang
    Katz, Evan
    Mascioli, Kirsten
    Chang, Kate
    Lockhart, David
    Valenzano, Kenneth
    [J]. MOLECULAR GENETICS AND METABOLISM, 2010, 99 (02) : S10 - S10
  • [3] Pharmacological chaperone therapy using migalastat: 1 year experience of starting a new therapy as reported by Fabry patients at a single UK centre
    Grillo, Giuseppina
    [J]. MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S64 - S64
  • [4] A review of gastrointestinal symptoms among patients affected with Fabry disease- One centre experience
    Green, Diane
    Ellis, Hannah
    Stepien, Karolina
    Jovanovic, Ana
    Sharma, Reena
    Wilcox, Gisela
    Thompson, Lorraine
    [J]. MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S64 - S64
  • [5] A PHARMACOGENETIC APPROACH TO THE SELECTION OF FABRY PATIENTS FOR PHARMACOLOGICAL CHAPERONE THERAPY
    Benjamin, E. R.
    Wu, X.
    Katz, E.
    Mascioli, K.
    Della Valle, M. C.
    Chang, H.
    Greene, D.
    Schiffmann, R.
    Lockhart, D. J.
    Valenzano, K. J.
    [J]. JOURNAL OF INHERITED METABOLIC DISEASE, 2010, 33 : S152 - S152
  • [6] A pharmacogenetic approach to predict response to pharmacological chaperone therapy for Fabry disease
    Benjamin, E. R.
    Wu, X.
    Khanna, R.
    Sitaraman, S. A.
    Palling, D. J.
    Schiffmann, R.
    Lockhart, D. J.
    Valenzano, K. J.
    [J]. JOURNAL OF INHERITED METABOLIC DISEASE, 2008, 31 : 128 - 128
  • [7] Mortality in Fabry disease cohort: One centre experience
    Thompson, Lorraine A.
    Stepien, Karolina M.
    Sharma, Reena
    [J]. MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S142 - S142
  • [8] A one tertiary centre experience: Is compliance with migalastat an issue in Fabry disease patients?
    Adrees, Faiza
    Wilcox, Gisela
    Sharma, Reena
    Stepien, Karolina
    Thompson, Lorraine
    Jovanovic, Ana
    [J]. MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S18 - S18
  • [9] Chaperone Therapy in Fabry Disease
    Weidemann, Frank
    Jovanovic, Ana
    Herrmann, Ken
    Vardarli, Irfan
    [J]. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2022, 23 (03)
  • [10] The effectiveness of vein mapping in reducing the number of missed infusions among Fabry disease patients: One centre experience
    Forshaw-Hulme, Stuart
    Jones, Mairead
    Gorton, Janet
    Sharma, Reena
    Wilcox, Gisela
    Stepien, Karolina
    [J]. MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S55 - S56