The Roche HD natural history study - An external comparator by design

被引：0
|
作者：
Hooper, G. [1 ]
Palermo, G. [2 ]
Hlavac, F. [3 ]
Finnegan, C. [4 ]
Frick, E. [5 ]
Boak, L. [6 ]
Doody, R. [7 ,8 ]
Schobel, S. [6 ]
Leavitt, B. [9 ]
机构：
[1] Roche Prod Ltd, Neurosci Huntingtons Dis, Welwyn Garden City, Herts, England
[2] F Hoffmann La Roche Ltd, Prod Dev, Basel, Switzerland
[3] F Hoffmann La Roche Ltd, Prod Dev Global, Basel, Switzerland
[4] Almac Grp, Almac Clin Technol LLC, Souderton, PA USA
[5] F Hoffmann La Roche Ltd, PD Clin Operat, Basel, Switzerland
[6] F Hoffmann La Roche Ltd, Prod Dev Neurosci, Basel, Switzerland
[7] F Hoffmann La Roche Ltd, Basel, Switzerland
[8] Genentech Inc, Neurodegenerat, San Francisco, CA 94080 USA
[9] Univ British Columbia, Dept Med Genet, Ctr Mol Med & Therapeut, Vancouver, BC, Canada
来源：
JOURNAL OF THE NEUROLOGICAL SCIENCES | 2019年 / 405卷
关键词：
D O I：
10.1016/j.jns.2019.10.1409
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
WCN19-1911
引用
收藏
页数：1
相关论文
共 50 条
  • [41] Natural history and quality of life study on muscular dystrophies in adulthood (Muskel-LIV): study design and conduct
    Nordstrom, S.
    Wahlgren, L.
    Lindberg, C.
    Tulinius, M.
    Sofou, K.
    NEUROMUSCULAR DISORDERS, 2024, 43
  • [42] Study design and baseline findings from the progression of ocular findings (PROOF) natural history study of dry eye
    McDonnell, Peter J.
    Pflugfelder, Stephen C.
    Stern, Michael E.
    Hardten, David R.
    Conway, Taryn
    Villanueva, Linda
    Hollander, David A.
    BMC OPHTHALMOLOGY, 2017, 17
  • [43] Progression of Ocular Findings (PROOF) Study of the Natural History of Dry Eye: Study Design and Baseline Patient Characteristics
    McDonnell, Peter
    Pflugfelder, Steve
    Schiffman, Rhett
    Hardten, David
    Stern, Michael
    Conway, Taryn
    Hollander, David
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2013, 54 (15)
  • [44] The natural history of multiple sclerosis: implications for trial design
    Wingerchuk, DM
    Weinshenker, BG
    CURRENT OPINION IN NEUROLOGY, 1999, 12 (03) : 345 - 349
  • [45] The TrialNet Natural History Study of the Development of Type 1 Diabetes: objectives, design, and initial results
    Mahon, Jeffrey L.
    Sosenko, Jay M.
    Rafkin-Mervis, Lisa
    Krause-Steinrauf, Heidi
    Lachin, John M.
    Thompson, Clinton
    Bingley, Polly J.
    Bonifacio, Ezio
    Palmer, Jerry P.
    Eisenbarth, George S.
    Wolfsdorf, Joseph
    Skyler, Jay S.
    PEDIATRIC DIABETES, 2009, 10 (02) : 97 - 104
  • [46] CENTRAL VEIN OCCLUSION STUDY - DESIGN, BASE-LINE, AND EARLY NATURAL-HISTORY
    FINKELSTEIN, D
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 1993, 34 (04) : 1442 - 1442
  • [47] Metachromatic leukodystrophy: from natural history study to the design of a protocol of ex vivo gene therapy
    Fumagalli, F.
    Biffi, A.
    Rovelli, A.
    Cesani, M.
    Del Carro, U.
    Baldoli, C.
    Roncarolo, M. G.
    Comi, G.
    Sessa, M.
    JOURNAL OF NEUROLOGY, 2009, 256 : S158 - S158
  • [48] The initiation, design, and establishment of the Desmoid Tumor Research Foundation Patient Registry and Natural History Study
    Mercier, Kelly A.
    Walsh, Darragh M.
    RARE TUMORS, 2019, 11
  • [49] TOPICS IN APPLIED GEOGRAPHY - THE BRANCH PLANT ECONOMY - A STUDY OF EXTERNAL CONTROL - WATTS,HD
    HUGHES, JT
    URBAN STUDIES, 1983, 20 (02) : 252 - 253
  • [50] A methodology study to evaluate external comparator arm study results versus randomized controlled trial results: Multiple myeloma case study
    Hoogendoorn, Lisette
    Sanz, Hector
    Quinten, Chantal
    Pignatti, Francesco
    Largent, Joan
    Rippin, Gerd
    PHARMACOEPIDEMIOLOGY AND DRUG SAFETY, 2022, 31 : 437 - 438
12345