5 Key Takeaways

• 1

  A retrospective study at Manchester Royal Eye Hospital evaluated long-term outcomes of Voretigene neparvovec-rzyl therapy.

• 2

  The study followed 13 eyes from 7 patients over an average of 22 months, focusing on biallelic RPE65 mutation.

• 3

  Patients showed improvements in light sensitivity, visual fields, and navigational ability, particularly in low-light conditions.

• 4

  Mean best-corrected visual acuity increased from 1.03 to 1.19 LogMAR, with significant improvements in full-field stimulus testing.

• 5

  Adverse events included vitritis, anterior uveitis, macular edema, and loss of the ellipsoid zone in some patients.