TreatSMA wish you good health and access to treatments in 2021.
2020 was a busy year for us as we continued to fight for access for children and adults to Spinraza, Zolgensma and the EAMS for Risdiplam. We have continued to post educational Webinars as a community resource – totalling 36 Webinars, you can watch these back on our YouTube channel https://www.youtube.
- We made our submission to Scottish Medicines Consortium on Zolgensma
- We campaigned and raised awareness within the community for EAMS for Risdiplam
- We represent families for NICE assessment of gene therapy for SMA and will also be involved with Risdiplam appraisal committee in 2021 – including having the first adult with SMA accepted onto the panel for the Risdiplam appraisal.
- We have continued to collect Type 3 evidence
- We have had a program of adult and children focused physiotherapy by Marion
- Community activities during lockdown including Bake Off, 80s Disco, Halloween Competition and a Message from Father Christmas
- We have offered a 24hr support and helpdesk service
- We set up the Fallen Petals support group
- We held a webinar on School and Covid with a SENDCO from a SEN school
- SMA Awareness Month where we held webinars with experts on various topics regarding SMA, posted daily infographics and posted videos from community members.
- We continued to put our surveys and collection of data to submit for various appraisals
- We continue to advocate for patients who do not have access to treatments, being vocal as part of managed agreement oversight committee or proactively contacting trusts with regards to EAMS and wide access to Spinraza.
- We continued to have meetings with NICE and MHRA
- We continued to campaign and push for NICE to review the MAA guidance for Spinraza for non ambulatory type 3s
- We continue to have interaction with Roche, Biogen and Novartis
- AA Diet support and meetings
- Marion joined the team
- Marni joined the team
- We provided birthday vouchers to those who had a birthday in the first full lockdown
- ‘What about us’ and ‘What If’ campaigns
- Covid support and guidance
- We Paired up with Annabelle Rose Foundation for food vouchers over Christmas
- Rare Disease Day event as well as attending and hosting a stall at Rare Disease Day
- Amongst other things we now also fully engaged into newborn screening topics to ensure that families get diagnosis quickly and access treatments much faster.
- Written letters of support for community members for various issues
- Remembered those who we have lost to SMA as well as Anita and those involved in The Jennifer Trust https://www.facebook.
All of this has been achieved by a very small team on a very small budget, it may be in the next year you see us pushing for fundraising a little more, but this is only so we can achieve what we would like to achieve for the community. We will continue to support and campaign for all treatment for ALL in 2021 as well as continue to engage with the community, inform through webinars and raise awareness for SMA. We wish you all a Happy New Year!