Avalglucosidase alfa improves motor and respiratory function in late-onset Pompe disease: A COMET win-ratio analysis

被引:0
|
作者
Diaz-Manera, Jordi [1 ]
Dimachkie, Mazen M. [2 ]
Ishak, Jack [3 ]
Caro, Jaime [4 ]
Hamed, Alaa [5 ]
Franca, Lionel Riou [6 ]
Thibault, Nathan [5 ]
Shukla, Pragya [3 ]
Berger, Kenneth I. [7 ]
Boentert, Matthias [8 ]
机构
[1] Newcastle Univ, John Walton Muscular Dystrophy Res Ctr, Ctr Life, Newcastle Upon Tyne, England
[2] Univ Kansas, Dept Neurol, Med Ctr, Kansas City, KS USA
[3] Evidera, Montreal, PQ, Canada
[4] Evidera, Waltham, MA USA
[5] Sanofi, Cambridge, MA USA
[6] Sanofi, Paris, France
[7] New York Univ, Div Pulm Crit Care & Sleep Med, Grossman Sch Med, New York, NY USA
[8] Munster Univ Hosp, Dept Neurol, Munster, Germany
关键词
D O I
10.1016/j.ymgme.2022.107076
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
93
引用
收藏
页码:34 / 34
页数:1
相关论文
共 50 条
  • [1] Avalglucosidase Alfa Improves Motor and Respiratory Function in Late-onset Pompe Disease: A COMET Win-Ratio Analysis
    Diaz-Manera, J.
    Ishak, J.
    Caro, J.
    Hamed, A.
    Franca, Riou L.
    Thibault, N.
    Shukla, P.
    Berger, K.
    Dimachkie, M.
    Boentert, M.
    NEUROMUSCULAR DISORDERS, 2022, 32
  • [2] Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease
    Boentert, Matthias
    Berger, Kenneth I.
    Diaz-Manera, Jordi
    Dimachkie, Mazen M.
    Hamed, Alaa
    Franca, Lionel Riou
    Thibault, Nathan
    Shukla, Pragya
    Ishak, Jack
    Caro, J. Jaime
    ORPHANET JOURNAL OF RARE DISEASES, 2024, 19 (01)
  • [3] Evaluating avalglucosidase alfa for the management of late-onset Pompe disease
    Angelini, Corrado
    EXPERT REVIEW OF NEUROTHERAPEUTICS, 2024, 24 (03) : 259 - 266
  • [4] Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease
    Matthias Boentert
    Kenneth I. Berger
    Jordi Díaz-Manera
    Mazen M. Dimachkie
    Alaa Hamed
    Lionel Riou França
    Nathan Thibault
    Pragya Shukla
    Jack Ishak
    J. Jaime Caro
    Orphanet Journal of Rare Diseases, 19
  • [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.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S152 - S152
  • [6] Efficacy and Safety Results of the Avalglucosidase alfa Phase 3 COMET Trial in Late-Onset Pompe Disease Patients
    Kushlaf, Hani
    Attarian, Shahram
    Borges, Joao Lindolfo
    Bouhour, Francoise
    Chien, Yin-Hsiu
    Choi, Young-Chul
    Clemens, Paula
    Day, John
    Diaz-Manera, Jordi
    Erdem-Ozdamar, Sevim
    Goker-Alpan, Ozlem
    Illarioshkin, Sergey
    Kishnani, Priya
    Kostera-Pruszczyk, Anna
    Ladha, Shafeeq
    Mozaffar, Tahseen
    Roberts, Mark
    Straub, Volker
    Toscano, Antonio
    van der Ploeg, Ans
    Haack, Kristina An
    Hug, Christopher
    Huynh-Ba, Olivier
    Zhou, Tianyue
    Johnson, Judith
    Dimachkie, Mazen
    Schoser, Benedikt
    NEUROLOGY, 2021, 96 (15)
  • [7] Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in late-onset Pompe disease patients
    Kishnani, Priya S.
    Attarian, Shahram
    Borges, Joao Lindolfo
    Bouhourd, Francoisee
    Chien, Yin-Hsiu
    Choi, Young-Chul
    Clemens, Paula
    Day, John
    Diaz-Manera, Jordi
    Erdem-Ozdamar, Sevim
    Goker-Alpan, Ozlem
    Illarioshkin, Sergey
    Kostera-Pruszczyk, Anna
    Kushlaf, Hani
    Ladha, Shafeeq
    Mozaffar, Tahseen
    Roberts, Mark
    Straub, Volker
    Toscano, Antonio
    van der Ploeg, Ans
    Haack, Kristina An
    Huynh-Ba, Olivier
    Zhou, Tianyue
    Dimachkie, Mazen
    Schoser, Benedikt
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S57 - S57
  • [8] COMET: Efficacy and safety of avalglucosidase alfa in late-onset Pompe disease participants after 97 weeks of treatment
    Schoser, B.
    Kishnani, P.
    Diaz-Manera, J.
    Kushlaf, H.
    Ladha, S.
    Mozaffar, T.
    Straub, V.
    Toscano, A.
    Van der Ploeg, A.
    Berger, K.
    Clemens, P.
    Chien, Y.
    Day, J.
    Ilarioshkin, S.
    Roberts, M.
    Attarian, S.
    Carvalho, G.
    Choi, Y.
    Erdem-Ozdamar, S.
    Goker-Alpan, O.
    Kostera-Pruszczyk, A.
    Haack, K. An
    Thibault, N.
    Zhou, T.
    Dimachkie, M.
    EUROPEAN JOURNAL OF NEUROLOGY, 2022, 29 : 59 - 60
  • [9] Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in late-onset Pompe disease patients
    Schoser, B.
    Attarian, S.
    Borges, J.
    Bouhour, F.
    Chien, Y.
    Choi, Y.
    Clemens, P.
    Day, J. W.
    Manera, J. Diaz
    Ozdamar, S. Erdem
    Goker-Alpan, O.
    Illarioshkin, S.
    Kishnani, P.
    Kostera-Pruszczyk, A.
    Kushlaf, H.
    Ladha, S.
    Mozaffar, T.
    Roberts, M.
    Straub, V.
    Toscano, A.
    Van der Ploeg, A.
    Haack, K. An
    Huynh-Ba, O.
    Zhou, T.
    Dimachkie, M.
    EUROPEAN JOURNAL OF NEUROLOGY, 2021, 28 : 68 - 68
  • [10] COMET post hoc analysis: efficacy of long-term avalglucosidase alfa in subgroups of patients with late-onset Pompe disease
    Toscano, A.
    Kishnani, P.
    Dimachkie, M.
    Sacconi, S.
    van der Beek, N.
    Roberts, M.
    Suwazono, S.
    Choi, Y.
    de Souza, P. Sgobbi
    Schoser, B.
    Armstrong, N.
    Huynh-Ba, O.
    Thibault, N.
    Periquet, M.
    Diaz-Manera, J.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S152 - S152