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Showing 1 to 10 of 10 results for nusinersen
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 [GID-TA11386] Expected publication date: 04 December 2024
Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in people of all ages.
Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies.
Managed access allows patients 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.
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.
NICE announces start of review of nusinersen Managed Access Agreement
NICE has today (29 October 2020) announced that it has begun the process to review data collected as part of the Managed Access Agreement (MAA) for nusinersen.
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NICE has today (2 June 2021) published draft guidance for which does not recommend risdiplam for treating the rare genetic disorder spinal muscular atrophy (SMA).
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.