A two-year prospective natural history study in patients with CRB1-associated retinal dystrophies: establishing clinical endpoints for future gene therapy trials

被引：0

作者：

Xuan-Thanh-An Nguyen ^{[1
]}

Talib, Mays ^{[1
]}

van Schooneveld, Mary J. ^{[2
]}

Wijnholds, Jan ^{[1
,3
]}

van Genderen, Maria M. ^{[4
,5
]}

Florijn, Ralph J. ^{[6
]}

ten Brink, Jacoline ^{[6
]}

Cremers, Frans P. ^{[7
,8
]}

Meester, Magda A. ^{[9
]}

Klaver, Caroline ^{[9
,10
]}

van den Born, L. Ingeborgh ^{[11
]}

Hoyng, Carel C. B. ^{[10
]}

Thiadens, Alberta ^{[9
]}

Bergen, Arthur ^{[3
,6
]}

Boon, Camiel ^{[1
,2
]}

机构：

[1] Leiden Univ, Med Ctr, Dept Ophthalmol, Leiden, Netherlands

[2] Univ Amsterdam, Dept Ophthalmol, Med Ctr, Amsterdam, Netherlands

[3] Netherlands Inst Neurosci, Amsterdam, Netherlands

[4] Univ Med Ctr Utrecht, Dept Ophthalmol, Utrecht, Netherlands

[5] Diagnost Ctr Complex Visual Disorders, Bartimeus, Zeist, Netherlands

[6] Univ Amsterdam, Dept Clin Genet, Med Ctr, Amsterdam, Netherlands

[7] Radboud Univ Nijmegen, Med Ctr, Dept Human Genet, Nijmegen, Netherlands

[8] Radboud Univ Nijmegen, Med Ctr, Donders Inst Brain Cognit & Behav, Nijmegen, Netherlands

[9] Erasmus MC, Dept Ophthalmol, Rotterdam, Netherlands

[10] Radboud Univ Nijmegen, Med Ctr, Dept Ophthalmol, Nijmegen, Netherlands

[11] Rotterdam Eye Hosp, Rotterdam, Netherlands

来源：

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2020年 / 61卷 / 07期

关键词：

D O I：

暂无

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

3051

引用

页数：4

共 21 条

[1] A two-year prospective natural history study in patients with CRB1-associated retinal dystrophies: Establishing clinical endpoints for future gene therapy trials
Nguyen, X. -T. -A.
Talib, M.
van Schooneveld, M. J.
Wijnhold, J.
van Genderen, M. M.
Florijn, R. J.
ten Brink, J. B.
Cremers, F. P. M.
Meester-Smoor, M. A.
Klaver, C. C. W.
van den Born, L. I.
Hoyng, C. B.
Thiadens, A. A. H. J.
Bergen, A. A.
Boon, C. J. F.
[J]. ACTA OPHTHALMOLOGICA, 2020, 98 : 22 - 22
[2] Extensive phenotyping in CRB1-associated retinal dystrophies: defining clinical endpoints for gene therapy trials
Talib, M.
van Schooneveld, M. J.
Wijnholds, J.
van Genderen, M.
Schalij-Delfos, N. E.
Florijn, R. J.
Cremers, F. P. M.
van den Born, L. I.
Thiadens, A. A.
Hoyng, C. B.
Klaver, C. C. W.
Bergen, A. A.
Boon, C. J. F.
[J]. ACTA OPHTHALMOLOGICA, 2018, 96 : 27 - 27
[3] Extensive phenotyping in CRB1-associated retinal dystrophies: defining clinical endpoints for gene therapy trials
Talib, Mays
van Schooneveld, Mary
Wijnholds, Jan
van Genderen, Maria M.
Schalij-Delofs, N.
Florijn, Ralph
Cremers, Frans P.
Born, Ingeborgh
Thiadens, Alberta A. H. J.
Hoyng, Carel C. B.
Klaver, Caroline C. W.
Bergen, Arthur A.
Boon, Camiel
[J]. INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2018, 59 (09)
[4] CRB1-Associated Retinal Dystrophies: A Anticipation of Future Clinical Trials
Nguyen, Xuan-thanh-an
Talib, Mays
van Schooneveld, Mary J.
Wijnholds, J. A. N.
van Genderen, Maria M.
Schalij-delfos, Nicoline E.
Klaver, Caroline C. W.
Talsma, Herman E.
Fiocco, Marta
Florijn, Ralph J.
ten Brink, Jacoline B.
Cremers, Frans P. M.
Meester-smoor, Magda A.
van den Born, L. Ingeborgh
Hoyng, Carel B.
Thiadens, Alberta A. H. J.
Bergen, Arthur A.
Boon, Camiel J. F.
[J]. AMERICAN JOURNAL OF OPHTHALMOLOGY, 2022, 234 : 37 - 48
[5] CRB1-Associated Retinal Dystrophies: Genetics, Clinical Characteristics, and Natural History
Varela, Malena Daich
Georgiou, Michalis
Alswaiti, Yahya
Kabbani, Jamil
Fujinami, Kaoru
Fujinami-Yokokawa, Yu
Khoda, Shaheeni
Mahroo, Omar A.
Robson, Anthony G.
Webster, Andrew R.
AlTalbishi, Alaa
Michaelides, Michel
[J]. AMERICAN JOURNAL OF OPHTHALMOLOGY, 2023, 246 : 107 - 121
[6] Defining inclusion criteria and endpoints for clinical trials: a prospective cross-sectional study in CRB1-associated retinal dystrophies
Talib, Mays
van Schooneveld, Mary J.
Wijnholds, Jan
van Genderen, Maria M.
Schalij-Delfos, Nicoline E.
Talsma, Herman E.
Florijn, Ralph J.
ten Brink, Jacoline B.
Cremers, Frans P. M.
Thiadens, Alberta A. H. J.
van den Born, L. Ingeborgh
Hoyng, Carel B.
Meester-Smoor, Magda A.
Bergen, Arthur A.
Boon, Camiel J. F.
[J]. ACTA OPHTHALMOLOGICA, 2021, 99 (03) : E402 - E414
[7] Four-year prospective follow-up in patients with CRB1-associated retinal dystrophies
Karuntu, J. S.
Nguyen, X.
Talib, M.
van Schooneveld, M. J.
Wijnholds, J.
van Genderen, M. M.
Schalij-Delfos, N. E.
Klaver, C. C. W.
Talsma, H. E.
Florijn, R. J.
ten Brink, J. B.
Meester-Smoor, M. A.
van den Born, L. I.
Hoyng, C. B.
Thiadens, A. A. H. J.
Bergen, A. A.
Boon, C. J. F.
[J]. ACTA OPHTHALMOLOGICA, 2022, 100 : 20 - 20
[8] Four-year prospective follow-up in patients with CRB1-associated retinal dystrophies
Karuntu, Jessica Serena
Nguyen, Xuan-Thanh-An
Talib, Mays
Van Schooneveld, Mary J.
Wijnholds, Jan
Van Genderen, Mies M.
Schalij-Delfos, Nicoline E.
Klaver, Caroline C. W.
Florijn, Ralph J.
Ten Brink, Jacoline B.
Meester-Smoor, Magda M.
Van den Born, L. Ingeborgh
Hoyng, Carel C. B.
Thiadens, Alberta A. H. J.
Bergen, Arthur A.
Boon, Camiel J. F.
[J]. INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2022, 63 (07)
[9] Quality of life in patients with CRB1-associated retinal dystrophies: A longitudinal study
Nguyen, X. -T. -A.
Karuntu, J. S.
Talib, M.
van Schooneveld, M. J.
Wijnholds, J.
van Genderen, M. M.
Schalij-Delfos, N. E.
Klaver, C. C. W.
Meester-Smoor, M. A.
van den Born, L. I.
Hoyng, C. B.
Thiadens, A. A. H. J.
Bergen, A. A.
van Nispen, R. M. A.
Boon, C. J. F.
[J]. ACTA OPHTHALMOLOGICA, 2023, 101 : 26 - 26
[10] Quality of life in patients with CRB1-associated retinal dystrophies: A longitudinal study
Karuntu, Jessica S.
Nguyen, Xuan-Thanh-An
Talib, Mays
van Schooneveld, Mary J.
Wijnholds, Jan
van Genderen, Maria M.
Schalij-Delfos, Nicoline E.
Klaver, Caroline C. W.
Meester-Smoor, Magda A.
van den Born, L. Ingeborgh
Hoyng, Carel B.
Thiadens, Alberta A. H. J.
Bergen, Arthur A.
van Nispen, Ruth M. A.
Boon, Camiel J. F.
[J]. ACTA OPHTHALMOLOGICA, 2024, 102 (04) : 469 - 477

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