SMA Newsroom

TreatmentsJul 25, 2023

Community update from Roche

We are pleased to share the latest community update from Roche, announcing that the CHMP (Committee for Medicinal Products for Human Use) at the European Medicines Agency (EMA) has recommended the approval of an indication extension for risdiplam (Evrysdi), for all ages, from birth.


TreatmentsJul 20, 2023

Community statement: BIOCAD

Biocad, a Russian biotechnology company, shares a community statement on its gene therapy product, ANB-004, which is being developed to treat SMA.

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.