SMA Newsroom

OdySMAJun 8, 2023

Publication of OdySMA abstract in the Orphanet Journal of Rare Diseases

We are delighted to see that the OdySMA abstract submitted after the European Conference on Rare Diseases (ECRD) 2022, was recently published in the Orphanet Journal of Rare Diseases on Friday, 2nd June.

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. (...)"