Long-term experience from an Expanded Access Program with idebenone in pediatric LHON patients

LHON results in bilateral, severe central vision loss, and is caused by mitochondrial DNA mutations. LHON is typically diagnosed between 15 - 30 years of age, although it can be detected earlier. Idebenone, the only approved treatment for LHON in Europe, has been shown to be efficacious and safe in a large proportion of adult patients, but pediatric data is limited. The Expanded Access Program (EAP, a named patient program under local regulations) provides insights into the potential of idebenone in pediatric LHON.