or nusinersen (intrathecal injection) administered before apparent signs of disease emerged. The study included presymptomatic infants with two or three copies of SMN2 at risk for developing SMA ...

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 ...

Cost-effectiveness body NICE has rejected Biogen’s Spinraza (nusinersen) therapy for the rare genetic muscle wasting disease spinal muscular atrophy (SMA) because of its high cost – but is in ...

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 ...

A wearable robotic device called HAL improved walking ability for SMA patients who also received Spinraza (nusinersen), a ...