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Guidance programme

Showing 1 to 9 of 9 results for risdiplam

  1. Nusinersen and risdiplam for treating spinal muscular atrophy (TA1162)

    Evidence-based recommendations on nusinersen (Spinraza) and risdiplam (Evrysdi) for treating spinal muscular atrophy.

  2. Intrathecal onasemnogene abeparvovec for treating spinal muscular atrophy in people 2 years and over [ID6556]

    In development Reference number: GID-TA11733 Expected publication date:  26 August 2026

  3. Forward view - our priority topics

    Our forward view highlights the areas we will prioritise in the coming year.

  4. Managed access

    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.

  5. Routine NHS access to spinal muscular atrophy treatments offers new hope for patients and families

    People living with spinal muscular atrophy (SMA) in England are set to gain routine access to two potentially life-changing treatments, following our publication of final draft guidance today.

  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. 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    NICE has today (2 June 2021) published draft guidance for which does not recommend risdiplam for treating the rare genetic disorder spinal muscular atrophy (SMA).