Risdiplam is an SMN2 pre-mRNA splicing modifier designed ... It's one of three drugs approved for SMA, the others being nusinersen (Spinraza) and onasemnogene abeparvovec (Zolgensma).

Zolgensma will compete against another highly expensive drug - Biogen's Spinraza (nusinersen), while Roche is also developing a potential rival called risdiplam that is slated for a European ...

Spinraza (nusinersen) – is one result of that effort. The antisense oligonucleotide works by modulating alternative splicing of the SMN2 exon which is mutated in the disease. This is however ...

Spinraza (nusinersen) is an injection therapy widely approved for ... Motor neurons are the specialized nerve cells that control voluntary movement. While humans also have SMN2, a so-called backup ...

A higher number of SMN2 copies is associated with milder SMA types. Spinraza (nusinersen), by Biogen, was the first disease-modifying therapy approved for all main SMA types. It is indicated for ...

They are very different in their trajectories in which symptoms tend to arise relative to many factors, but especially their SMN2 copy number when patients are first diagnosed. At the end of the ...

Delandistrogene moxeparvovec for treating Duchenne muscular dystrophy in children 4 to 7 years ID3897 Technology appraisal guidance TBC Guselkumab for previously treated moderately to severely active ...

Guselkumab for previously treated moderately to severely active Crohn's disease ID6238 Technology appraisal guidance TBC Nusinersen and risdiplam for treating spinal muscular atrophy (review of TA588 ...

Nusinersen significantly improved the motor function of the patients especially those with SMA type 1. “Our findings support early SMA screening and presymptomatic nusinersen administration to ...