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

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 compa...

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Detalles Bibliográficos
Autores principales: Romagnoli, Martina, La Morgia, Chiara, Carbonelli, Michele, Di Vito, Lidia, Amore, Giulia, Zenesini, Corrado, Cascavilla, Maria Lucia, Barboni, Piero, Carelli, Valerio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Brief Communications
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7187718/
https://www.ncbi.nlm.nih.gov/pubmed/32243103
http://dx.doi.org/10.1002/acn3.51026
Descripción
Sumario: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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