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

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 ...

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

NVS reports new safety and efficacy data from the phase III program for its investigational OAV101 IT gene therapy for SMA ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

Developed by Ireland-based BrePco Biopharma, Neoatricon is the first paediatric-strength dopamine hydrochloride infusion for ...

Novartis also shared results from the open-label phase 3b STRENGTH study of OAV101 IT in SMA patients aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam, which ...