Zeleciment rostudirsen (z-rostudirsen)

The DELIVER registrational expansion cohort included 32 participants, with 24 allocated to z-rostudirsen and 8 to placebo. At six months, mean absolute dystrophin was 5.46% of normal after muscle-content adjustment and 2.87% without adjustment. A post-hoc functional comparison used 21 treated participants and 18 pooled placebo participants. Reported differences were 0.04 rise/sec for TTR velocity and 0.2 m/sec for 10MWR velocity, both with nominal p<0.05; the NSAA difference was 2.1 points with nominal p=0.0894. No related serious TEAEs were reported in the registrational cohort. Related serious events occurred in extension participants, and higher-dose events included acute kidney injury, thrombocytopenia, and pancytopenia.