Zolgensma data 'demonstrate the value of newborn screening for SMA'

Swiss pharma giant Novartis (NOVN: VX) has announced new data that reinforce the benefit of Zolgensma (onasemnogene abeparvovec), an essential one-time gene therapy treatment that has been approved in many markets to treat spinal muscular atrophy (SMA).

The completed Phase III SPR1NT study demonstrated that children with three copies of the SMN2 back-up gene who were treated presymptomatically achieved age-appropriate motor milestones, including standing and walking.

"Results from SPR1NT again confirm the remarkable impact of Zolgensma for children at risk for SMA who are treated before the onset of symptoms"In addition, a descriptive post-hoc analyses of START, STR1VE-EU and STR1VE-US indicated that children with SMA Type 1 achieved or maintained important measures of bulbar function following treatment with Zolgensma, including the ability to speak, swallow, meet nutritional needs and maintain airway protection.