Skottland ger det gröna ljus till Spinraza, men endast för SMA-typ 1

SMC har rekommenderat att denna behandling endast finansieras för barn med SMA-typ 1. Detta är ett positivt steg framåt och en livlinje för dessa barn och familjer. Det är mycket nedslående att barn, ungdomar och vuxna med SMA typ 2 eller typ 3 inte har tillgång till behandling och kan dra nytta av det

1 kommentar

  1. Mia

    Biogen’s statement on the SMC decision

    Patients with type 1 SMA are already receiving treatment via the Biogen expanded access programme (EAP), which provides infantile onset SMA patients (consistent with type 1) with nusinersen, free of charge. Now that the SMC has recommended nusinersen for these patients, the EAP will close in Scotland and they will transition to treatment provided by NHS Scotland.

    Biogen is in active discussions with the SMC to resubmit nusinersen for later onset SMA (types 2 and 3) and urges the initiation of a reformed appraisal process for ultra-orphan medicines as soon as possible to allow a greater degree of flexibility for the assessment of such treatment options. These reforms were proposed to the Scottish Government in 2016 following a review of the system and the government confirmed that they would take forward the recommendations. Biogen hopes this new, more appropriate process will lead to a positive outcome and we remain steadfast in our commitment to making nusinersen available to all who may benefit.

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