Advocacy for Access to Drug Treatments for SMA

Since Autumn 2016, we have invested a huge amount of time and energy to advocate for access to all three treatments (nusinersen, zolgensma and risdiplam) in the UK, as well as joining with others to voice the need for reform of regulatory systems and processes.

Our work on the specific drug treatments for SMA has been with the Community, other patient groups and clinicians. Together, we have made a substantial impact that has over time led to positive decisions by NICE, NHS England and the Scottish Medicines Consortium (SMC). Our work is by no means finished as we continue to monitor the roll-out of these treatments, working with individuals needing access as well as with Trusts, NHS England and NICE. We are also now part of the UK SMA Newborn Screening Alliance working for the inclusion of SMA in the newborn heel prick blood test.

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