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Full field stimulus testing (FST) to assess sepofarsen patient response in Leber congenital amaurosis type 10

ePoster

Published on November 13, 2020 at AAO

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Author(s)

Allen Ho1, Artur V Cideciyan2, Samuel G Jacobson2, Stephen R Russell3, Arlene V Drack3, Bart P Leroy4, 5, 6, Michael R Schwartz7, Wilhelmina den Hollander7, Hsin Yi Huang van Eekelen7, Agathe Plichta7, Friedrich Asmus7, Gerard Platenburg7, Aniz Girach7, David M Rodman7

1 Wills Eye Hospital, Thomas Jefferson University, Philadelphia, Pennsylvania, USA; 2 Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA; 3 The University of Iowa Institute for Vision Research, University of Iowa, Iowa, USA; 4 Center for Medical Genetics, Ghent University Hospital, Ghent, Belgium; 5 Department of Ophthalmology, Ghent University and Ghent University Hospital, Ghent, Belgium; 6 Division of Ophthalmology and Center for Cellular Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; 7 ProQR Therapeutics, Leiden, the Netherlands.

Description

In a phase 1b/2 trial, 11 LCA10 patients received intravitreal sepofarsen. The goal was to evaluate patient response to sepofarsen through assessment of visual acuity and full-field stimulus testing (FST). Data show that FST seems a more sensitive method in patients with severely impaired vision.

Recognized with best poster award at AAO 2020.

AAO 2020 poster - FST to assess sepofarsen patient response in LCA10

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Tags

  • AAO
  • Leber Congenital Amaurosis
  • Sepofarsen

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Related publications

  • Results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene

    AAO 2020 poster by Stephen Russel et al. Recognized with best poster award. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.

    Date: November 13, 2020

    Author(s): Russell SR, Drack AV, et al.

    ePoster
  • Results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene

    ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.

    Date: June 17, 2020

    Author(s): Russell SR, Drack AV, et al.

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  • ARVO (2021) Russell SR et al., Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension trial in Leber Congenital Amaurosis type 10 (LCA10)

    ARVO 2021 presentation by Stephen R. Russell from Iowa University on the Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension trial in CEP290 mediated Leber Congenital Amaurosis type 10.

    Date: May 06, 2021

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