Idebenone increases chance of stabilization/recovery of visual acuity in  OPA1 ‐dominant optic atrophy | Zendy

Romagnoli Martina | Zendy; La Morgia Chiara | Zendy; Carbonelli Michele | Zendy; Di Vito Lidia | Zendy; Amore Giulia | Zendy; Zenesini Corrado | Zendy; Cascavilla Maria Lucia | Zendy; Barboni Piero | Zendy; Carelli Valerio | Zendy

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Idebenone increases chance of stabilization/recovery of visual acuity in OPA1 ‐dominant optic atrophy

Author(s) -

Romagnoli Martina,

La Morgia Chiara,

Carbonelli Michele,

Di Vito Lidia,

Amore Giulia,

Zenesini Corrado,

Cascavilla Maria Lucia,

Barboni Piero,

Carelli Valerio

Publication year - 2020

Publication title -

annals of clinical and translational neurology

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.824

H-Index - 42

ISSN - 2328-9503

DOI - 10.1002/acn3.51026

Subject(s) - medicine , idebenone , visual acuity , atrophy , confounding , cohort , ophthalmology

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.

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