A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a ...

Next, he underwent a catheter targeted dynamic myelogram by directed intrathecal enhancement tomography (DIET) technique, which detected a focal ventral epidural leak at the level of C6 left of the ...

An intravenous form of the gene therapy is already approved and sold as Zolgensma in the US for patients less than 2 years of age.

Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...

Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal ...

Novartis has reported positive efficacy and safety outcomes from its Phase III clinical programme for OAV101 IT (onasemnogene ...