Clinical benefits of miglustat on bone disease in adult type 1 Gaucher disease (GD1): A meta-analysis

被引:0
|
作者
Pastores, G. M.
Elstein, D.
Hrebicek, M.
Zimran, A.
机构
[1] NYU, Sch Med, Dept Neurol & Pediat, New York, NY USA
[2] Shaare Zedek Med Ctr, Dept Med, IL-91000 Jerusalem, Israel
[3] Inst IMD, Prague, Czech Republic
关键词
D O I
暂无
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:S68 / S68
页数:1
相关论文
共 50 条
  • [21] Multimorbidity in type 1 Gaucher disease patients under miglustat therapy
    Engay, Blanca Medrano
    Roy, Ana
    Atutxa, Koldo
    Diaz-Morant, Vicente
    Garcia-Frade, Javier
    Hernandez-Martin, Roberto
    Luno, Elisa
    Noya, Soledad
    de la Serna, Javier
    Villalon, Lucia
    Giraldo, Pilar
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S93 - S94
  • [22] Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials
    Deborah Elstein
    Nadia Belmatoug
    Patrick Deegan
    Özlem Göker-Alpan
    Derralynn A. Hughes
    Ida Vanessa D. Schwartz
    Neal Weinreb
    Nicola Bonner
    Charlotte Panter
    Donna Fountain
    Andrew Lenny
    Louise Longworth
    Rachael Miller
    Koonal Shah
    Jörn Schenk
    Rohini Sen
    Ari Zimran
    Orphanet Journal of Rare Diseases, 17
  • [23] Biomarker responses to eliglustat, an investigational oral substrate reduction therapy (SRT) for Gaucher disease type 1 (GD1)
    Peterschmitt, M. Judith
    Lukina, Elena
    Watman, Nora
    Dragosky, Marta
    Pastores, Gregory
    Avila Arreguin, Elsa
    Rosenbaum, Hanna
    Zimran, Ari
    Aguzzi, Rasha
    Angell, Jennifer
    Puga, Ana Cristina
    Cox, Gerald
    MOLECULAR GENETICS AND METABOLISM, 2013, 108 (02) : S74 - S74
  • [24] Effect of miglustat on bone disease in adults with type 1 Gaucher disease:: A pooled analysis of three multinational, open-label studies
    Pastores, Gregory M.
    Elstein, Deborah
    Hrebicek, Martin
    Zimran, Ari
    CLINICAL THERAPEUTICS, 2007, 29 (08) : 1645 - 1654
  • [25] Erratum to: Management of Bone Disease in Gaucher Disease Type 1: Clinical Practice
    Gaetano Giuffrida
    Maria Domenica Cappellini
    Francesca Carubbi
    Maja Di Rocco
    Giovanni Iolascon
    Advances in Therapy, 2015, 32 : 388 - 389
  • [26] Correlating liver stiffness with disease severity scoring system (DS3) values in Gaucher disease type 1 (GD1) patients
    Serai, Suraj D.
    Naidu, Anjani P.
    Burrow, T. Andrew
    Prada, Carlos E.
    Xanthakos, Stavra
    Towbin, Alexander J.
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (03) : 357 - 363
  • [27] Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients
    Delbini, Paola
    Ghiandai, Viola
    Cappellini, Maria Domenica
    Duca, Lorena
    Nava, Isabella
    Cassinerio, Elena
    Motta, Irene
    BLOOD, 2019, 134
  • [28] BIOMARKER RESPONSES AFTER 3 YEARS OF TREATMENT WITH ELIGLUSTAT, AN INVESTIGATIONAL ORAL THERAPY FOR GAUCHER DISEASE TYPE 1 (GD1)
    Puga, A. C.
    Lukina, E.
    Watman, N.
    Dragosky, M.
    Iastrebner, M.
    Pastores, G.
    Avila Arreguin, E.
    Phillips, M.
    Rosenbaum, H.
    Aguzzi, R.
    Peterschmitt, M. J.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2011, 34 : S202 - S202
  • [29] ELIGLUSTAT, AN INVESTIGATIONAL ORAL THERAPY FOR GAUCHER DISEASE TYPE 1 (GD1): PHASE 2 RESULTS AFTER 3 YEARS
    Puga, A. C.
    Lukina, E.
    Watman, N.
    Dragosky, M.
    Iastrebner, M.
    Pastores, G.
    Avila Arreguin, E.
    Phillips, M.
    Rosenbaum, H.
    Aguzzi, R.
    Bertin-Millet, D.
    Peterschmitt, M. J.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2011, 34 : S201 - S201
  • [30] ELIGLUSTAT, AN INVESTIGATIONAL ORAL THERAPY FOR GAUCHER DISEASE TYPE 1 (GD1): PHASE 2 RESULTS AFTER 4 YEARS
    Puga, A. C.
    Lukina, E.
    Dragosky, M.
    Iastrebner, M.
    Pastores, G.
    Avila Arreguin, E.
    Zimran, A.
    Rosenbaum, H.
    Watman, N.
    Sysoeva, E.
    Aguzzi, R.
    Ross, L. H.
    Peterschmitt, M. J.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2012, 35 : S98 - S98