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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)

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Published on May 06, 2021 at ARVO

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Author(s)

Stephen R. Russell1, Arlene V. Drack1, Artur V. Cideciyan2, Samuel G. Jacobson2, Bart P. Leroy3,4, Wilhelmina den Hollander5, Adriana Hollestein-Havelaar5, Michael R. Schwartz5, Aniz Girach5

1Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, IA, USA; 2Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; 3Department of Ophthalmology & Center for Medical Genetics, Ghent University Hospital & Ghent University, Ghent, Belgium; 4Ophthalmic Genetics & Visual Electrophysiology, Division of Ophthalmology, The Children’s Hospital of Philadelphia, PA, USA; 5ProQR Therapeutics, Leiden, the Netherlands

Tags

  • ARVO
  • 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

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

    AAO 2020 poster by Allen Ho et al. Recognized with best poster award. Data show that FST seems a more sensitive method in patients with severely impaired vision.

    Date: November 13, 2020

    Author(s): Ho A, Cideciyan AV, et al.

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  • 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
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