Novartis Poised for Phase 3b SMART Study of Zolgensma

Novartis Poised for Phase 3b SMART Study of Zolgensma

293092

Novartis Poised for Phase 3b SMART Study of Zolgensma

Novartis will initiate SMART, a Phase 3b clinical study to evaluate the safety and efficacy of the one-time gene therapy Zolgensma (onasemnogene abeparvovec) in young children with spinal muscular atrophy (SMA) who weigh 8.5 to 21 kilograms (about 18 to 46 pounds).  Zolgensma is approved in the U.S. to treat all types of SMA in newborns and toddlers up to age 2. However, according to the Pediatric Neuromuscular Clinical Research’s (PNCR) natural history study of…

You must be logged in to read/download the full post.