Spinal Muscular Atrophy (SMA) SMA is a rare and devastating genetic disease caused by a lack of a functional survival motor neuron 1 (SMN1) gene, resulting in the rapid and irreversible loss of motor …

Nov 24, 2025 · At Novartis, we're doubling down on our commitment to neurology, expanding our legacy of innovation in spinal muscular atrophy (SMA) and multiple sclerosis (MS) to work in …

Basel, March 20, 2023 — Novartis today presented new data which underscore the transformational and sustained benefit of Zolgensma ® (onasemnogene abeparvovec), an essential one-time gene …

Dec 30, 2024 · Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle …

Mar 4, 2024 · The SMART study is the first open-label clinical study of Zolgensma to include previously treated patients 1-6 Basel, March 4, 2024 – Novartis today presented new data that continue to …

Mar 19, 2025 · Novartis plans to file applications with regulatory agencies in H1 2025 Basel, March 19, 2025 – Novartis announced positive safety and efficacy results from the Phase III program for …

Basel, May 24, 2019 - AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma ® (onasemnogene abeparvovec-xioi) for the …

Intrathecal Onasemnogene Abeparvovec (OAV101 IT) for Treatment-Experienced Patients with Spinal Muscular Atrophy: Phase 3b, Open-Label STRENGTH Study

In its most severe forms, spinal muscular atrophy (SMA) can progress rapidly. But the early signs can sometimes be subtle, and may even go unseen for weeks or months as the disease progresses. …

ITVISMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with …