Author Archives: Steve Bryson PhD

Major Developmental Milestones Met in Infants, Zolgensma Data Show

New data about Zolgensma, the one-time gene therapy for children with spinal muscular atrophy (SMA), demonstrate age-appropriate development when used pre-symptomatically, and rapid, meaningful efficacy in symptomatic children, even those with severe disease before treatment, according to two Phase 3 clinical trials. The therapy, developed by Novartis, uses…

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.

Quantitative MRI Is Sensitive Measure of Muscle Decline

Young adults with spinal muscular atrophy (SMA) exhibited thigh muscle decline despite stable strength and motor function over one year, a pilot study using quantitative MRI imaging demonstrated.  The scientists said this non-invasive muscle imaging technique shows its potential as a…