Hand-out
Press Releases
New Novartis Phase III data demonstrate meaningful efficacy and safety results of intrathecal onasemnogene abeparvovec in broad patient population with SMA
Treatment with investigational OAV101 IT led to statistically significant 2.39-point improvement on the HFMSE vs. 0.51 points in sham control armSafety findings were consistent in both treatment-naïve and treatment-experienced patientsThese
