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Guidance programme

Showing 1 to 11 of 11 results for nusinersen

  1. Nusinersen for treating spinal muscular atrophy (TA588)

    Evidence-based recommendations on nusinersen (Spinraza) for treating spinal muscular atrophy in children and adults.

  2. Nusinersen and risdiplam for treating spinal muscular atrophy (review of TA588 and TA755) [ID6195]

    In development [GID-TA11386] Expected publication date: 04 December 2024

  3. Risdiplam for treating spinal muscular atrophy (TA755)

    Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in people of all ages.

  4. Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)

    Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies.

  5. Managed access

    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.

  6. 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.

  7. NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy

    NICE has today (2 June 2021) published draft guidance for public consultation which does not recommend risdiplam (also called Evrysdi and made by Roche) for treating the rare genetic disorder spinal muscular atrophy (SMA).

  8. NICE announces more people eligible for nusinersen following review of Managed Access Agreement

    NICE has today (4 May 2021) announced that more people with the rare genetic disorder spinal muscular atrophy (SMA) are to benefit from nusinersen (also called Spinraza and made by Biogen) following a review of data collected as part of the Managed Access Agreement (MAA).

  9. 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 (also called Spinraza and made by Biogen) for treating the rare genetic disorder spinal muscular atrophy (SMA).

  10. NICE final draft guidance approves life-changing gene therapy for treating spinal muscular atrophy

    A new and potentially curative 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.

  11. NICE draft guidance recommends new treatment for spinal muscular atrophy as part of a managed access agreement

    Around 1,500 people in England with the rare genetic disorder spinal muscular atrophy are set to benefit from a new treatment after NICE today (19 November 2021) published draft guidance recommending risdiplam (also called Evrysdi and made by Roche) as part of a managed access agreement (MAA).