SMA Newsroom

TreatmentsMar 21, 2025

Community update from Novartis: Phase III STEER and Phase IIIb STRENGTH studies

Novartis shared updates on the results from Phase III STEER and Phase IIIb STRENGTH studies, evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in people living with SMA aged two to <18 years. 

TreatmentsDec 20, 2024

Community Update Biohaven: RESILIENT Letter to SMA Community

Biohaven released the results from their RESILIENT SMA clinical trial, and they did not meet the primary endpoint. Biohaven is currently analysing the data further and is engaging in discussion with Health Authorities. 

TreatmentsDec 20, 2024

Community Update Roche: End of Year Community Letter

We are pleased to share the latest Community Letter issued by Roche, providing an update on their ongoing efforts in Spinal Muscular Atrophy (SMA) in 2024. The letter includes the latest developments on risdiplam and Roche’s SMA clinical development program.