Long-term functional and structural outcomes in X-linked retinoschisis: implications for clinical trials
Beau J. Fenner; Jonathan F. Russell; Arlene V. Drack; Alina V. Dumitrescu; Elliott H. Sohn; Stephen R. Russell; H. Culver Boldt; Louisa M. Affatigato · 2023 · Frontiers in Medicine
WASTE classifies this as Negative / Null Result Report · AI classification, approximate
The study found no significant effect — useful as a negative control or null benchmark for your own design.
Abstract
Introduction X-linked retinoschisis (XLRS) is an inherited retinal disease (IRD) caused by pathogenic mutations in the retinoschisin gene, RS1 . Affected individuals develop retinal layer separation, leading to loss of visual acuity (VA). Several XLRS gene therapy trials have been attempted but none have met their primary endpoints. An improved understanding of XLRS natural history and clinical outcomes may better inform future trials. Here, we report the long-term functional and structural outcomes of XLRS and the relevance of RS1 genotypes to the visual prognosis of affected individuals. Met
Abstract by Beau J. Fenner; Jonathan F. Russell; Arlene V. Drack; Alina V. Dumitrescu; Elliott H. Sohn; Stephen R. Russell; H. Culver Boldt; Louisa M. Affatigato, Frontiers in Medicine (2023) — licensed CC BY 4.0.
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Metadata source: OpenAlex · DOI 10.3389/fmed.2023.1204095