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This article is the author's final published version in American Journal of Ophthalmology Case Reports, Volume 32, 2023, Article number 101873.

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Copyright © 2023 The Authors. Published by Elsevier Inc.

This is an open access article under the CC BY-NC-ND license ( nc-nd/4.0/).


PURPOSE: An intravitreally injected antisense oligonucleotide, sepofarsen, was designed to modulate splicing within retinas of patients with severe vision loss due to deep intronic c.2991 + 1655A > G variant in the

OBSERVATIONS: Visual function was evaluated with best corrected standard and low-luminance visual acuities, microperimetry, dark-adapted chromatic perimetry, and full-field sensitivity testing. Retinal structure was evaluated with OCT imaging. At the fovea, all visual function measures and IS/OS intensity of the OCT showed transient improvements peaking at 3-6 months, remaining better than baseline at ∼2 years, and returning to baseline by 3-4 years after each single injection.

CONCLUSIONS AND IMPORTANCE: These results suggest that sepofarsen reinjection intervals may need to be longer than 2 years.

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This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.

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