SRP-9003 Efficacy, Tolerability Sustained at 2 Years in LGMD2E
SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores at 2 years in patients with limb-girdle muscular dystrophy Type 2E.
New Data Showcase Onasemnogene Abeparvovec Efficacy in SMA
Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.