Novartis announced new long-term data for Zolgensma® (onasemnogene abeparvovec).

The latest data from two Long-Term Follow-Up (LTFU) studies, LT-001 and LT-002, show the continued efficacy and durability of Zolgensma across a range of patient populations, with an overall benefit-risk profile that remains favourable, with treated children maintaining motor milestones up to 7.5 years post-dosing.

Today we want to celebrate with you the official launch of our OdySMA project. OdySMA is an initiative that aims at one and only goal: that no one is left behind in the access to treatment journey.

OdySMA is a dynamic tool that we are using to create an SMA atlas that visualises systematically collected data to illustrate access pathways. The goal is to reveal the 'quest to access' of people living with SMA by mapping, visualising and centralising knowledge and data around access issues.

Novartis Gene Therapies has just recently issued a community update about two acute liver failure cases in Russia and Kazakhstan reported back in August 2022.