Impact of endpoint selection on sample size calculations for disease-modifying clinical trials in Parkinson's disease patients with GBA1 mutations

被引:0
|
作者
Simon, David K. [1 ]
Mendonca, Nuno [2 ]
Holenz, Joerg [3 ]
Liu, Jennifer [1 ]
Castilla-Fernandez, Guillermo [2 ]
Fonseca, Miguel M. [3 ]
机构
[1] Beth Israel Deaconess Med Ctr, Boston, MA USA
[2] Bial R&D Investments SA, Coronado, Portugal
[3] Bial Portela & Ca SA, Coronado, Portugal
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
POSTER 28
引用
收藏
页码:S10 / S11
页数:2
相关论文
共 50 条
  • [41] Mild cognitive impairment among LRRK2 and GBA1 patients with Parkinson's disease
    Thaler, Avner
    Livne, Vered
    Rubinstein, Einat
    Omer, Nurit
    Faust-Socher, Achinoam
    Cohen, Batsheva
    Giladi, Nir
    Shirvan, Julia C.
    Cedarbaum, Jesse M.
    Gana-Weisz, Mali
    Goldstein, Orly
    Orr-Urtreger, Avi
    Alcalay, Roy N.
    Mirelman, Anat
    PARKINSONISM & RELATED DISORDERS, 2024, 123
  • [42] Clinical manifestation of patients with Parkinson's disease carriers of "severe" mutations in the GBA gene
    Rozenberg, E.
    Mirelman, A.
    Levy, S.
    Kedmi, M.
    Orr-Urtreger, A.
    Giladi, N.
    Gurevich, T.
    MOVEMENT DISORDERS, 2010, 25 (07) : S473 - S473
  • [43] Patient Diaries As a Clinical Endpoint in Parkinson's Disease Clinical Trials
    Papapetropoulos, Spyridon
    CNS NEUROSCIENCE & THERAPEUTICS, 2012, 18 (05) : 380 - 387
  • [44] Neuroimaging biomarkers for clinical trials of disease-modifying therapies in Alzheimer's disease
    Dickerson B.C.
    Sperling R.A.
    NeuroRX, 2005, 2 (2): : 348 - 360
  • [45] The Role of Cholesterol in α-Synuclein and Lewy Body Pathology in GBA1 Parkinson's Disease
    Garcia-Sanz, Patricia
    M.F.G. Aerts, Johannes
    Moratalla, Rosario
    MOVEMENT DISORDERS, 2021, 36 (05) : 1070 - 1085
  • [46] Consensus Guidance for Genetic Counseling in GBA1 Variants: A Focus on Parkinson's Disease
    Vieira, Sophia R. L.
    Mezabrovschi, Roxana
    Toffoli, Marco
    Del Pozo, Sara Lucas
    Menozzi, Elisa
    Mullin, Stephen
    Yalkic, Selen
    Limbachiya, Naomi
    Koletsi, Sofia
    Loefflad, Nadine
    Lopez, Grisel J.
    Gan-Or, Ziv
    Alcalay, Roy N.
    Sidransky, Ellen
    Schapira, Anthony H. V.
    MOVEMENT DISORDERS, 2024, 39 (12) : 2144 - 2154
  • [47] PPMI driven sample size estimation for clinical trials in Parkinson's disease
    Marek, K.
    Seibyl, J.
    Caspell-Garcia, C.
    Coffey, C.
    Mollenhauer, B.
    Kieburtz, K.
    Tanner, C.
    Chahine, L.
    Siderowf, A.
    Simuni, T.
    MOVEMENT DISORDERS, 2018, 33 : S18 - S18
  • [48] Secretome Analyses Identify FKBP4 as a GBA1-Associated Protein in CSF and iPS Cells from Parkinson's Disease Patients with GBA1 Mutations
    Kojima, Rika
    Paslawski, Wojciech
    Lyu, Guochang
    Arenas, Ernest
    Zhang, Xiaoqun
    Svenningsson, Per
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2024, 25 (01)
  • [49] Clinicogenetic study of GBA mutations in patients with familial Parkinson's disease
    Li, Yuanzhe
    Sekine, Takeshi
    Funayama, Manabu
    Li, Lin
    Yoshino, Hiroyo
    Nishioka, Kenya
    Tomiyama, Hiroyuki
    Hattori, Nobutaka
    NEUROBIOLOGY OF AGING, 2014, 35 (04) : 935.e3 - 935.e8
  • [50] A newly implemented NGS-based method to detect GBA1 variants in patients with Parkinson's disease
    Monfrini, E.
    Palmieri, I.
    Cuconato, G.
    Percetti, M.
    Cortelli, P.
    Di Fonzo, A.
    Valente, E. M.
    MOVEMENT DISORDERS, 2023, 38 : S484 - S484