The treatment almost completely halted the decline in the 6-minute walk test (6MWT) proving the drug is very effective. This is GREAT progress. The two that had to discontinue treatment reinforces the critical need to begin treatment as early as possible. This makes an incredibly strong case to get the FDAs accelerated approval, as patients deprived of the treatment may eventually degrade to a point where nothing helps - compared with early treatment that almost completely neutralizes the effect of Duchenne muscular dystrophy (DMD).
These results are impressive in every way. Safe too.