Biogen Inc. (NASDAQ:BIIB), which licensed the rights to nusinersen from Ionis Pharmaceuticals, stated that the new regimen could offer meaningful benefits to patients. The higher dose comprises ...

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

Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

The approval of nusinersen, an RNA-based drug that restores muscle function when injected into the spine, marked a significant milestone in the field of gene therapy. Since then, thousands of ...

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

A higher dose of Spinraza may be more effective than the currently approved dosing schedule for maintaining motor function in ...

The open-label STRENGTH study assessed the safety, tolerability, and efficacy of OAV101 IT in 27 patients with SMA who had previously discontinued nusinersen or risdiplam. Results found that the total ...

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 ...

For example, Biogen’s nusinersen, an ASO therapy for the fatal neuromuscular condition spinal muscular atrophy (SMA), can reduce by half the likelihood of death or the need for mechanical ...