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Showing 1 to 7 of 7 results for risdiplam
Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in people of all ages.
Nusinersen and risdiplam for treating spinal muscular atrophy (review of TA588 and TA755) [ID6195]
In development Reference number: GID-TA11386 Expected publication date: TBC
In development Reference number: GID-TA11733 Expected publication date: 26 August 2026
Managed access allows people to access promising new treatments that would otherwise not be recommended because it is too uncertain whether the treatment is cost-effective.
Our forward view highlights the topics we will prioritise in the coming year.
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy (HST24)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating presymptomatic spinal muscular atrophy in babies aged 12 months and under.
Risdiplam not recommend for treating spinal muscular atrophy
NICE has today (2 June 2021) published draft guidance for which does not recommend risdiplam for treating the rare genetic disorder spinal muscular atrophy (SMA).