Question about what happens with the current trial patients
I was wondering if the current trial patients will still be able to continue taking the drug or not during a Phase III trial? Will they automatically be enrolled in the next phase or will they possibly become ineligible?
I am really upset that the FDA is playing with these kids' lives. I really hope the boys who are showing improvement in Phase 2b will be able to continue the drug going forward.
The boys in the current trial get to take the drug as long as they want. I think the company has to formally extend the trial - they mentioned on the call that going forward they will only be getting 6MWT and other data every 6 months, so the next data point will be 120 weeks.