The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have accepted Biogen’s applications for a higher dose regimen of spinal muscular atrophy (SMA) treatment Spinraza ...

Spinal muscular atrophy (SMA) therapy Spinraza has been a key earner for Biogen in recent years, but with sales now heading into reverse, the company is looking to extend its franchise.

In 2016, Spinraza became the first approved treatment for SMA, with exceptional results in infants with severe SMA – so exceptional, it was considered unethical to continue enrolling children ...

As its name suggests, splicing involves snipping out certain sequences in the RNA called ... disorder spinal muscular atrophy (SMA) – Spinraza (nusinersen) – is one result of that effort.

The firm expanded its neurology portfolio beyond MS, including the blockbuster Spinraza for spinal muscular atrophy, or SMA, a rare neuromuscular disease. We see Biogen as a firm in transition ...