We are pleased to share with you the latest community update issued by Roche.
It contains the information shared at the Cure SMA Research & Clinical Care Meeting 2023 and Family Conference which took place last week.
Scholar Rock announces new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with apitegromab.
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.