Safety of home-based infusion of alglucosidase alfa in late-onset Pompe disease: 13 years of experience from the Erasmus MC university medical center

被引：0
|
作者：
Ditters, I. [1 ]
van Kooten, H. [2 ]
van der Beek, N. [2 ]
van den Hout, J. [1 ]
Huidekoper, H. [1 ]
van der Ploeg, A. [1 ]
机构：
[1] Sophia Childrens Univ Hosp, Erasmus MC, Rotterdam, Netherlands
[2] Erasmus MC, Rotterdam, Netherlands
来源：
NEUROMUSCULAR DISORDERS | 2021年 / 31卷
关键词：
D O I：
10.1016/j.nmd.2021.07.227
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
EP.202
引用
收藏
页码：S111 / S111
页数：1
相关论文
共 33 条
  • [1] Safety of home-based infusion of alglucosidase alfa in late onset Pompe disease: 13 years of experience from the Erasmus MC University Medical Center
    Ditters, Imke A. M.
    Van Kooten, Harmke
    van der Beek, Nadine A. M. E.
    van den Hout, Hannerieke
    Huidekoper, Hidde H.
    van der Ploeg, Ans T.
    [J]. MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S39 - S39
  • [2] Alglucosidase alfa: 5 years of experience in late-onset Pompe disease
    Benedikt Schoser
    [J]. BMC Musculoskeletal Disorders, 14 (Suppl 2)
  • [3] Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions
    Ditters, Imke A. M.
    van Kooten, Harmke A. A.
    van der Beek, Nadine A. M. E.
    Hardon, Jacqueline F. F.
    Ismailova, Gamida
    Brusse, Esther
    Kruijshaar, Michelle E. E.
    van der Ploeg, Ans T. T.
    van den Hout, Johanna M. P.
    Huidekoper, Hidde H. H.
    [J]. BIODRUGS, 2023, 37 (05) : 685 - 698
  • [4] Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions
    Imke A. M. Ditters
    Harmke A. van Kooten
    Nadine A. M. E. van der Beek
    Jacqueline F. Hardon
    Gamida Ismailova
    Esther Brusse
    Michelle E. Kruijshaar
    Ans T. van der Ploeg
    Johanna M. P. van den Hout
    Hidde H. Huidekoper
    [J]. BioDrugs, 2023, 37 : 685 - 698
  • [5] COMET: Safety of avalglucosidase alfa in patients with late-onset Pompe disease who switched treatment from alglucosidase alfa
    Diaz-Manera, J.
    Kishnani, P.
    Ladha, S.
    Miossec, P.
    Armstrong, N.
    Thibault, N.
    Periquet, M.
    Tammireddy, S.
    Dimachkie, M.
    Schoser, B.
    [J]. NEUROMUSCULAR DISORDERS, 2023, 33 : S152 - S152
  • [6] Efficacy and safety of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease: PROPEL study
    Kishnani, Priya
    Bratkovic, Drago
    Byrne, Barry
    Diaz-Manera, Jordi
    Laforet, Pascal
    Mozaffar, Tahseen
    van der Ploeg, Ans
    Roberts, Mark
    Toscano, Antonio
    Jiang, Hai
    Sitaraman, Sheela
    Goldman, Mitchell
    Castelli, Jeff
    Schoser, Benedikt
    [J]. GENETICS IN MEDICINE, 2022, 24 (03) : S96 - S96
  • [7] Home-Based Electrical Impedance Myography in Late-Onset Pompe Disease
    Hobson-Webb, Lisa
    Zwelling, Paul
    Pifer, Ashley
    Kishnani, Priya
    [J]. NEUROLOGY, 2020, 94 (15)
  • [8] Safety analysis of home-based enzyme replacement therapy with alglucosidase alfa in Pompe disease: A prospective study
    Ditters, Imke A. M.
    van der Ploeg, Ans T.
    van der Beek, Nadine A. M. E.
    van den Hout, Hannerieke . M. P.
    Huidekoper, Hidde H.
    [J]. MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 35 - 36
  • [9] Safety analysis of home-based enzyme replacement therapy with alglucosidase alfa in Pompe disease; a prospective study
    Ditters, I.
    van der Ploeg, A.
    van der Beek, N.
    van den Hout, J.
    Huidekoper, H.
    [J]. NEUROMUSCULAR DISORDERS, 2022, 32 : S77 - S77
  • [10] EFFICACY AND SAFETY OF CIPAGLUCOSIDASE ALFA/MIGLUSTAT VERSUS ALGLUCOSIDASE ALFA/PLACEBO IN LATE-ONSET POMPE DISEASE: A PHASE 3 TRIAL (PROPEL)
    Byrne, Barry J.
    Bratkovic, Drago
    Laforet, Pascal
    Mozaffar, Tahseen
    van der Ploeg, Ans
    Roberts, Mark
    Schoser, Benedikt
    Toscano, Antonio
    Jiang, Hai
    Sitaraman, Sheela
    Kuchipudi, Srilakshmi
    Kazi, Zoheb
    Goldman, Mitchell
    Castelli, Jeff
    Kishnani, Priya S.
    [J]. MUSCLE & NERVE, 2021, 64 : S64 - S65
1234