Shots:
The US FDA has granted IND clearance to initiate a P-I multiple ascending dose trial assessing DYNE-302 against PBO in ambulatory adults with facioscapulohumeral muscular dystrophy (FSHD)
In the first cohort, 9 pts will receive DYNE-302 (1.5 mg/kg, Q4W) or PBO, with higher doses & less frequent dosing planned. Pts completing the PBO-controlled phase…
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…

