What we are campaining to make any and all treatments to SMA available to everyone with the disorder.
In particular, we want to make sure that Spinraza is appraised by NICE and is then offered by the NHS in the entire spectrum of SMA, across all SMA types, ages, and functional abilities.
To this aim, we are reaching out, both as individual families and as a Community, to the decision makers. We tell them what SMA is all about and that even minor functional improvements mean a lot to SMA patients and their carers.
In January 2017, we took part in the Spinraza scoping workshop at NICE, the agency responsible for appraising all treatments in the country. Throughout the winter and spring of 2017, we worked towards making the Spinraza Expanded Access Programme possible in the UK, all whilst the country lagged behind most of the world in starting the programme. We wrote to MPs, we linked up with hospitals across the country, we held meetings with managers and directors at NHS. As a result, and thanks to an involvement of SMA medical experts and other SMA charities, EAP became a reality in the UK this same spring.
In July 2017, we presented the SMA cause at a meeting at the House of Lords.
Throughout the 2018 we engaged in the Spinraza appraisal process, participating in a series of meetings with health authorities in which we always pushed for a wide availability of Spinraza. We also engaged with the MPs as well as reached out to the regulators.
In September, in response to the negative NICE recommendation, we organised protests in London and Manchester.
We are currently putting our our resources to the fight for the only existing effective treatment for SMA.