Patients in the Pompe Registry who switched from alglucosidase alfa to avalglucosidase alfa: Real-world experience

被引：0
|
作者：
Schoser, B. [1 ]
Abbott, M. [2 ]
Toscano, A. [3 ]
Foster, M. [4 ]
Periquet, M. [5 ]
Sparks, S. [4 ]
Kishnani, P. [6 ]
机构：
[1] LMU Klinikum Munchen, Dept Neurol, Friedrich Baur Inst, Munich, Germany
[2] Baystate Hlth, Springfield, MA USA
[3] Univ Messina, Dept Clin & Expt Med, Reference Ctr Rare Neuromuscular Disorders, Messina, Italy
[4] Sanofi, Cambridge, MA USA
[5] Sanofi, Berlin, Germany
[6] Duke Univ, Med Ctr, Durham, NC USA
来源：
EUROPEAN JOURNAL OF NEUROLOGY | 2023年 / 30卷
关键词：
D O I：
暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
EPO-394
引用
收藏
页码：572 / 573
页数：2
相关论文
共 50 条
  • [41] Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry
    Kishnani, Priya S.
    Kronn, David
    Suwazono, Shugo
    Broomfield, Alexander
    Llerena, Juan
    Al-Hassnan, Zuhair Nasser
    Batista, Julie L.
    Wilson, Kathryn M.
    Periquet, Magali
    Daba, Nadia
    Hahn, Andreas
    Chien, Yin-Hsiu
    [J]. ORPHANET JOURNAL OF RARE DISEASES, 2023, 18 (01)
  • [42] Real-World Use of Octocog Alfa and Damoctocog Alfa Pegol in Women with Hemophilia a from the ATHNdataset
    Chandler, Martin
    Charlet, Jessica
    Moulton, Thomas
    Recht, Michael
    [J]. BLOOD, 2022, 140 : 2751 - 2752
  • [43] Real-world utilization of darbepoetin alfa in cancer chemotherapy patients
    Pan, Xiaoyun
    Nordstrom, Beth L.
    MacLachlan, Sharon
    Lin, Junji
    Xu, Hairong
    Sharma, Anjali
    Chandler, David
    Li, Xiaoyan
    [J]. JOURNAL OF ONCOLOGY PHARMACY PRACTICE, 2019, 25 (01) : 16 - 24
  • [44] Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
    Tim A. Kanters
    Ans T. van der Ploeg
    Michelle E. Kruijshaar
    Dimitris Rizopoulos
    W. Ken Redekop
    Maureen P. M. H. Rutten-van Mӧlken
    Leona Hakkaart-van Roijen
    [J]. Orphanet Journal of Rare Diseases, 12
  • [45] Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
    Kanters, Tim A.
    van der Ploeg, Ans T.
    Kruijshaar, Michelle E.
    Rizopoulos, Dimitris
    Redekop, W. Ken
    Rutten-van Molken, Maureen P. M. H.
    Hakkaart-van Roijen, Leona
    [J]. ORPHANET JOURNAL OF RARE DISEASES, 2017, 12
  • [46] AVALGLUCOSIDASE ALFA (AVA) IMPROVES SYMPTOMS AND FUNCTIONING IN LATE-ONSET POMPE DISEASE (LOPD) PATIENTS VS ALGLUCOSIDASE ALFA (ALG): POST-HOC ANALYSES OF PATIENT-REPORTED OUTCOMES (PROS) FROM COMET TRIAL
    Toscano, A.
    Van der Pioeg, A. T.
    Berger, K., I
    MM, Dimachkie
    Schoser, B.
    Gwaitney, C.
    Msihid, J.
    Hamed, A.
    Thibault, N.
    Pollissard, L.
    Kishnani, P. S.
    [J]. VALUE IN HEALTH, 2022, 25 (12) : S403 - S403
  • [47] Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
    Dimachkie, Mazen M.
    Barohn, Richard J.
    Byrne, Barry
    Goker-Alpan, Ozlem
    Kishnani, Priya S.
    Ladha, Shafeeq
    Laforet, Pascal
    Mengel, Karl Eugen
    Pena, Loren D. M.
    Sacconi, Sabrina
    Straub, Volker
    Trivedi, Jaya
    Van Damme, Philip
    van der Ploeg, Ans T.
    Vissing, John
    Young, Peter
    Haack, Kristina An
    Foster, Meredith
    Gilbert, Jane M.
    Miossec, Patrick
    Vitse, Olivier
    Zhou, Tianyue
    Schoser, Benedikt
    [J]. NEUROLOGY, 2022, 99 (05) : E536 - E548
  • [48] Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline
    Kronn, David
    Davison, James
    Brassier, Anais
    Broomfield, Alexander
    Hahn, Si Houn
    Kumada, Satoko
    Labarthe, Francois
    Ohki, Hirotaka
    Prakalapakorn, S. Grace
    Haack, Kristina An
    Meng, Xianzhang
    Sparks, Susan
    Tammireddy, Swathi
    Wilson, Catherine
    Zaher, Atef
    Zhou, Tianyue
    Chien, Yin-Hsiu
    Kishnani, Priya S.
    [J]. MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S68 - S68
  • [49] Real-World Experience of Ropeginterferon-Alfa Treatment of PV and ET - Two Centers Experience
    Tashi, Tsewang
    Reeves, Brandi N.
    Kim, Soo Jin
    Jeong, Hyewon
    Chen, Sheh-Li
    Parsegov, Brynn
    Prchal, Josef T.
    [J]. BLOOD, 2023, 142
  • [50] Real-world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe
    Blatny, Jan
    Nielsen, Emma Munk
    Reitzel, Signe Baattrup
    McMillan, Annabell Cajus
    Dano, Anne
    Bystricka, Linda
    Kragh, Nana
    Klamroth, Robert
    [J]. HAEMOPHILIA, 2023, 29 (04) : 963 - 974
12345