CRB1-Associated Retinal Dystrophies: A Anticipation of Future Clinical Trials: A Prospective Natural History Study in Anticipation of Future Clinical Trials

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CRB1-Associated Retinal Dystrophies: A Anticipation of Future Clinical Trials: A Prospective Natural History Study in Anticipation of Future Clinical Trials
 
Nguyen, Xuan Thanh An; Talib, Mays; van Schooneveld, Mary J.; Wijnholds, Jan; 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.
(2022) American Journal of Ophthalmology, volume 234, pp. 37 - 48
(Article)
Abstract
PURPOSE: To investigate the natural disease course of retinal dystrophies associated with crumbs cell polarity complex component 1 (CRB1) and identify clinical end points for future clinical trials. DESIGN: Single-center, prospective case series. METHODS: An investigator-initiated nationwide collaborative study that included 22 patients with CRB1-associated retinal dystrophies. Patients underwent ophthalmic ... read more
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Keywords: Electroretinography, Eye Proteins/genetics, Humans, Membrane Proteins/genetics, Nerve Tissue Proteins/genetics, Retina, Retinal Dystrophies/diagnosis, Retinitis Pigmentosa/diagnosis, Tomography, Optical Coherence/methods, Visual Field Tests, Visual Fields, Journal Article, Research Support, Non-U.S. Gov't
DOI: https://doi.org/10.1016/j.ajo.2021.07.021
ISSN: 0002-9394
Publisher: Elsevier USA
Note: Funding Information: Funding/Support: This research was supported by Algemene Nederlandse Vereniging ter Voorkoming van Blindheid (ANVVB), Landelijke Stichting voor Blinden en Slechtzienden (LSBS), and the Oogfonds, which contributed through UitZicht (Delft, the Netherlands), as well as the Curing Retinal Blindness Foundation, Stichting Blindenhulp, Bontius Stichting, and Retina Fonds. These funding organizations had no role in the design or conduct of this research. Funding Information: Funding/Support: This research was supported by Algemene Nederlandse Vereniging ter Voorkoming van Blindheid (ANVVB), Landelijke Stichting voor Blinden en Slechtzienden (LSBS), and the Oogfonds, which contributed through UitZicht (Delft, the Netherlands), as well as the Curing Retinal Blindness Foundation, Stichting Blindenhulp, Bontius Stichting, and Retina Fonds. These funding organizations had no role in the design or conduct of this research. Financial Disclosures: Leiden University Medical Center is the holder of patent application PCT/NL2014/050549, which describes the potential clinical use of CRB2. Jan Wijnholds is listed as inventor on this patent and is an employee of Leiden University Medical Center. The other authors indicate no financial support or conflicts of interest. All authors attest that they meet the current ICMJE criteria for authorship. Acknowledgments:: L. Ingeborgh van den Born, Carel B. Hoyng, and Camiel J.F. Boon are members of the European Reference Network for Rare Eye diseases (ERN-EYE). Publisher Copyright: © 2021 The Authors Copyright © 2021 The Authors. Published by Elsevier Inc. All rights reserved.
(Peer reviewed)