Three-Year Safety Results of SAR422459 (EIAV-ABCA4) Gene Therapy in Patients With ABCA4-Associated Stargardt Disease: An Open-Label Dose-Escalation Phase I/IIa Clinical Trial, Cohorts 1-5.

Purpose: To report on the safety of the first 5 cohorts of a gene therapy trial using recombinant equine infectious anemia virus expressing ABCA4 (EIAV-ABCA4) in adults with Stargardt dystrophy due to mutations in ABCA4.

Design: Nonrandomized multicenter phase I/IIa clinical trial.

Methods: Patients received a subretinal injection of EIAVABCA4 in the worse-seeing eye at 3 dose levels and were followed for 3 years after treatment.

The 'dark' side of sedation: 12 years of office-based pediatric deep sedation for electroretinography in the dark.

Objectives: Analyze pediatric ERG data for adverse events, interventions, and outcomes of propofol sedations performed in near-complete darkness.

Aim: To demonstrate that deep sedation with propofol for ERG can be performed efficiently and safely in children in near-total darkness.

Background: Full-field electroretinography (ERG) is a valuable tool for the diagnosis of vision loss in children.