Shots:Japan's MHLW has granted conditional & time-limited approval to Elevidys for DMD pts (3–8yrs.) with no exon 8/9 deletions in the DMD gene & who are negative for anti-AAVrh74 AbsApproval was based on extensive clinical data, incl. 2yr. global P-III (EMBARK) trial showing motor function benefits vs external controls; Part 2 data was…
Shots:The P-III (EMBARK: N=126) study was carried out in 2 parts, with P1 involving ambulatory DMD boys (n=63; 4-7yrs.) to receive Elevidys (1.33x10^14 vg/kg) vs PBO while P2 involved crossing over of the patients (5-9yrs.); 59 crossed over to Elevidys post 52wks.
Parts 1 (post 2yrs.) & 2 (post 1yr.) depicted improved NSAA…

