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

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 ...

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 ...

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 ...