The next step in my research is a clinical trial for patients with choroideremia and Usher syndrome using the drug Translarna, which we aim to start in the next year. If successful, this therapy will have far reaching effects and will provide a potential treatment for a wide range of patients with inherited eye disease caused by this particular type of genetic mutation.
https://www.moorfieldseyecharity.org.uk/novel-treatments-incurable-inherited-conditions