Shots:
Novartis has reported data from the biomarker cohort of the P-I/II (FORTITUDE) study assessing delpacibart braxlosiran (2mg/kg, Q6W) vs PBO in 90 pts (16-70yrs.) with facioscapulohumeral muscular dystrophy (FSHD)
Trial met its 1 & key 2EPs, showing reductions in KHDC1L (cDUX) & creatine kinase biomarker levels, which indicates potent target engagement & decrease in…
