Real-world Zolgensma data reinforces transformational profile

Swiss pharma giant Novartis (NOVN: VX) has announced new data that reinforces the transformational benefit of Zolgensma (onasemnogene abeparvovec), a one-time gene therapy treatment for spinal muscular atrophy (SMA).

Children with SMA treated pre-symptomatically achieved age-appropriate motor milestones including sitting, standing and walking, required no ventilatory or feeding tube support and had no serious, treatment-related adverse events.

"Consistent, significant and clinically meaningful therapeutic benefit in SMA"Real-world data indicate older children, aged from six months, achieved clinically-meaningful benefit with Zolgensma alone, after or in combination with another SMA therapy, with events consistent with the previously described safety profile.