SMA Newsroom

TreatmentsMay 4, 2023

New interim data on long-term efficacy and safety data up to 7.5 years post-dosing of Zolgensma.

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.

AdvocacyApr 16, 2023

Rare Revolution Magazine: interview with Nicole Gusset.

Today we are very happy to share with you the interview with our CEO, Nicole Gusset, published by Rare Revolution Magazine.

Nicole shares her vision of SMA Europe and speaks about different initiatives led by our organisation:

"(...) Initially, we were all united in the same goal—all fighting for the same unmet need—lack of treatments. Now we have medicines, but these are not universally available across Europe (or indeed globally). We need to be careful in our work that we stay united. (...)"

Consensus for SMN2 genetic analysis in SMA patients at 270th ENMC International Workshop.

At the 270th ENMC International Workshop, which took place from 10 - 12th of March 2023 in the Netherlands, participants agreed to determine a standard operating procedure for SMN2 gene copy number determination and a specific workflow for diagnosis and management of SMA patients identified in neonatal screening as well as to engage collaborative efforts for the creation of a European network to address discrepancies among expert centres.