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.
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 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. (...)"
How does caring for a child with SMA impact the lives of their parents? What influences the parents’ well-being, both in a positive and negative way?