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Showing 1 to 11 of 11 results for nusinersen for treating spinal muscular atrophy
Evidence-based recommendations on nusinersen (Spinraza) for treating spinal muscular atrophy in children and adults.
Nusinersen and risdiplam for treating spinal muscular atrophy (review of TA588 and TA755) [ID6195]
In development Reference number: GID-TA11386 Expected publication date: TBC
Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies.
In development Reference number: GID-TA11733 Expected publication date: 26 August 2026
Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in people of all ages.
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.
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.
Our forward view highlights the topics we will prioritise in the coming year.
NICE approves life-changing gene therapy for treating spinal muscular atrophy
A new one-off gene therapy for babies with the rare genetic disorder spinal muscular atrophy (SMA) is set to become the most expensive treatment ever approved by NICE.
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NICE has today (2 June 2021) published draft guidance for which does not recommend risdiplam for treating the rare genetic disorder spinal muscular atrophy (SMA).
More people with rare genetic disorder eligible for nusinersen
Following a review of data collected as part of the Managed Access Agreement, more people with spinal muscular atrophy (SMA) are to benefit from nusinersen.