Clinical UncertaintyOne patient experienced a transient, reversible renal biomarker elevation, indicating that the program might not be out of the woods yet.
CompetitionPEPG is behind competitors in development, and it remains unclear if an accelerated path will be open.
Safety RisksThe safety profile still needs de-risking due to potential readthrough from the discontinued DMD program, where asymptomatic cases of hypomagnesemia were observed.