Eteplirsen's supporters argued on Monday that even a slight demonstrated clinical benefit should justify marketing approval given the lack of alternatives and the serious practical difficulties of developing therapies for rare diseases. Critics retorted that Sarepta's data and trial design was at fault. "We would like to thank the hundreds of patients and families who participated in the discussion today, underscoring the critical unmet need of people living with Duchenne," said Sarepta interim CEO Edward Kaye in a statement. "We appreciated the opportunity to present our data to the advisory committee panel and will continue to work with FDA as they complete their review of the eteplirsen NDA. Today more than ever, we remain committed to our mission of bringing a treatment to the Duchenne community."