Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene abeparvovec, ...

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

The SMA gene therapy candidate OAV101 IT, a version of Zolgensma, can be safely given to previously treated patients, per new ...

Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a ...

The drug, code-named OAV101, shares the same active ingredient as Zolgensma, which in 2019 gained U.S. approval for the ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

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

Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene abeparvovec, OAV101 IT, in a broad population of patients aged ...

OAV101 IT is an investigational gene replacement therapy designed to directly address the genetic root cause of the disease by replacing the nonworking SMN1 gene with a single dose.