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 ...

Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

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

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

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 ...

Results from the STEER and STRENGTH studies showed that OAV101 IT led to a 2.39-point improvement in motor function in patients with spinal muscular atrophy. Results from the Phase III STEER and Phase ...

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.