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

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

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

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

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

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 ...

Scholar Rock , a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases where protein growth ...

2 But this indication leaves out those older than 2 years, who are currently limited to non–gene therapy options like nusinersen (Spinraza; Biogen) and risdiplam (Evrysdi; Genentech), and in ...

Primary endpoint showed clinically meaningful improvement in patients with SMA receiving apitegromab as measured by gold standard Hammersmith Functional Motor Scale Expanded (HFMSE) versus placebo ...

For instance, in infants and children treated with the antisense oligonucleotide nusinersen, NfL levels in CSF were normalized following treatment, coinciding with notable motor function improvements.