FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

Biogen Inc. (NASDAQ:BIIB), which licensed the rights to nusinersen from Ionis Pharmaceuticals, stated that the new regimen ...

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 ...

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 case report in Japan shows Spinraza helped a boy with SMA type 0 sit without support for a short time at age 2 and stand ...

0.51 points in the sham control arm (P=0.0074).1-5 In the phase IIIb STRENGTH study, treatment with OAV101 IT in patients who have discontinued treatment with nusinersen or risdiplam demonstrated ...