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Guidance programme

Showing 1 to 6 of 6 results for risdiplam

  1. Risdiplam for treating spinal muscular atrophy (TA755)

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

  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. 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.

  4. 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.

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 NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy

    NICE has today (2 June 2021) published draft guidance for public consultation which does not recommend risdiplam (also called Evrysdi and made by Roche) for treating the rare genetic disorder spinal muscular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

  6. NICE draft guidance recommends new treatment for spinal muscular atrophy as part of a managed access agreement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disorder spinal muscular atrophy are set to benefit from a new treatment after NICE today (19 November 2021) published draft guidance recommending risdiplam (also called Evrysdi and made by Roche) as part of a managed access agreement (MAA).