Shots:
The US FDA has received an NDA for ecopipam, a selective dopamine D1 receptor antagonist, for the treatment of pediatric Tourette syndrome
NDA was supported by the P-III trial data showing that ecopipam significantly delayed time to relapse vs PBO in pediatric Tourette syndrome pts who responded to treatment during the open-label treatment period;…
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 Abs
Approval was based on extensive clinical data, incl. 2yr. global P-III (EMBARK) trial showing motor function benefits vs external controls; Part 2 data was…

