Advances in Drug Therapy for Usher Syndrome
Jennifer J. Lentz Usher Syndrome Family Conference July 11, 2015 Ø Overall goal
Ø Usher syndrome update and current hypotheses
Ø New therapeu c approaches
Ø Read-through small molecules
Ø An sense oligonucleo des (ASOs)
Ø Future Direc ons Overall Goal for the scien fic and medical Usher community
Ø Develop tools that provide a medical benefit to every individual with Usher syndrome Usher Syndrome Update
• Prevalence – Autosomal, recessive, gene