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- Title
Idebenone increases chance of stabilization/recovery of visual acuity in OPA1‐dominant optic atrophy.
- Authors
Romagnoli, Martina; La Morgia, Chiara; Carbonelli, Michele; Di Vito, Lidia; Amore, Giulia; Zenesini, Corrado; Cascavilla, Maria Lucia; Barboni, Piero; Carelli, Valerio
- Abstract
We previously documented that idebenone treatment in OPA1‐Dominant Optic Atrophy (OPA1‐DOA) led to some degrees of visual improvement in seven patients. We here present the results of a cohort study, which investigated the effect of off‐label idebenone administration in a larger OPA1‐DOA group compared with untreated patients. Inclusion criteria were: OPA1‐DOA clinical and molecular diagnosis, baseline visual acuity (VA) greater than/equal to counting fingers and treatment duration greater than 7 months. We found a significant difference between the last visit and baseline VA in favor of stabilization/recovery in idebenone‐treated as compared to untreated patients. This effect was retained after controlling for confounders.
- Subjects
VISUAL acuity; ATROPHY; MOLECULAR diagnosis; TREATMENT duration; COHORT analysis
- Publication
Annals of Clinical & Translational Neurology, 2020, Vol 7, Issue 4, p590
- ISSN
2328-9503
- Publication type
Article
- DOI
10.1002/acn3.51026