Two successfully completed pregnancies in adult onset Pompe disease, under continued treatment with alglucosidase alfa

被引：0
作者：
Julie Van Houtte
Jan L. De Bleecker
机构：
[1] Ghent University Hospital,Department of Neurology and Neuromuscular Reference Centre
来源：
Acta Neurologica Belgica | 2019年 / 119卷
关键词：
Pompe disease; Enzyme replacement therapy; Pregnancy; Myopathy;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
引用
收藏
页码：147 / 149
页数：2
相关论文
共 41 条
  • [31] STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa
    Kristina Gutschmidt
    Olimpia Musumeci
    Jordi Díaz-Manera
    Yin-Hsiu Chien
    Karl Christian Knop
    Stephan Wenninger
    Federica Montagnese
    Alessia Pugliese
    Graziana Tavilla
    Jorge Alonso-Pérez
    Paul Wuh-Liang Hwu
    Antonio Toscano
    Benedikt Schoser
    Journal of Neurology, 2021, 268 : 2482 - 2492
  • [32] Adult onset glycogen storage disease type II (adult onset Pompe disease): report and magnetic resonance images of two cases
    Andrew Del Gaizo
    Sima Banerjee
    Michael Terk
    Skeletal Radiology, 2009, 38 : 1205 - 1208
  • [33] Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial
    Toscano, Antonio
    Pollissard, Laurence
    Msihid, Jerome
    van der Beek, Nadine
    Kishnani, Priya S.
    Dimachkie, Mazen M.
    Berger, Kenneth I.
    Dasmahapatra, Pronabesh
    Thibault, Nathan
    Hamed, Alaa
    Zhou, Tianyue
    Haack, Kristina An
    Schoser, Benedikt
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
  • [34] 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.
    BIODRUGS, 2023, 37 (05) : 685 - 698
  • [35] Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial
    Schoser, Benedikt
    Roberts, Mark
    Byrne, Barry J.
    Sitaraman, Sheela
    Jiang, Hai
    Laforet, Pascal
    Toscano, Antonio
    Castelli, Jeff
    Diaz-Manera, Jordi
    Goldman, Mitchell
    Van der Ploeg, Ans T.
    Bratkovic, Drago
    Kuchipudi, Srilakshmi
    Mozaffar, Tahseen
    Kishnani, Priya S.
    LANCET NEUROLOGY, 2021, 20 (12) : 1027 - 1037
  • [36] Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naive and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study
    Pena, Loren D. M.
    Barohn, Richard J.
    Byrne, Barry J.
    Desnuelle, Claude
    Goker-Alpan, Ozlem
    Ladha, Shafeeq
    Laforet, Pascal
    Mengel, Karl Eugen
    Pestronk, Alan
    Pouget, Jean
    Schoser, Benedikt
    Straub, Volker
    Trivedi, Jaya
    Van Damme, Philip
    Vissing, John
    Young, Peter
    Kacena, Katherine
    Shafi, Raheel
    Thurberg, Beth L.
    Culm-Merdek, Kerry
    van der Ploeg, Ans T.
    NEUROMUSCULAR DISORDERS, 2019, 29 (03) : 167 - 186
  • [37] Population Pharmacokinetic Modeling and Determination of Individual Exposure to Avalglucosidase Alfa in Adolescent and Adult Patients With Late-Onset Pompe Disease: Analysis of Pooled Data From Phase I to III Clinical Trials
    Tuffal, Gilles
    Tiraboschi, Gilles
    Hurbin, Fabrice
    Boittet, Pascale
    Palmer, Rachel
    Martinez, Jean-Marie
    Fabre, David
    THERAPEUTIC DRUG MONITORING, 2023, 45 (05) : 644 - 652
  • [38] Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
    Kishnani, Priya S.
    Byrne, Barry J.
    Claeys, Kristl G.
    Diaz-Manera, Jordi
    Dimachkie, Mazen M.
    Kushlaf, Hani
    Mozaffar, Tahseen
    Roberts, Mark
    Schoser, Benedikt
    Hummel, Noemi
    Kopiec, Agnieszka
    Holdbrook, Fred
    Shohet, Simon
    Toscano, Antonio
    JOURNAL OF PATIENT-REPORTED OUTCOMES, 2024, 8 (01)
  • [39] Acid phosphatase-positive globular inclusions is a good diagnostic marker for two patients with adult-onset Pompe disease lacking disease specific pathology
    Tsuburaya, Rie S.
    Monma, Kazunari
    Oya, Yasushi
    Nakayama, Takahiro
    Fukuda, Tokiko
    Sugie, Hideo
    Hayashi, Yukiko K.
    Nonaka, Ikuya
    Nishino, Ichizo
    NEUROMUSCULAR DISORDERS, 2012, 22 (05) : 389 - 393
  • [40] Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials
    Mozaffar, Tahseen
    Fransa, Lionel Riou
    Shukla, Pragya
    Proskorovsky, Irina
    Zhou, Tianyue
    Periquet, Magali
    Haack, Kristina An
    Pollissard, Laurence
    Straub, Volker
    MOLECULAR GENETICS AND METABOLISM REPORTS, 2024, 40
12345