Genetic testing hopes to speed up drug development in rare eye diseases
Other treatment option is disfiguring eyelid operation
When Fabrizio Sottile went blind at the age of 17, he thought his dreams of becoming a world-class swimmer were over. But he wasn’t about to let Leber’s hereditary optic neuropathy (LHON) stand in his way, and he made his first appearance as part of Italy’s Paralympic team in 2016, aged 23.
US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.