January – February 2018

We invited people to complete our on-line surveys via: a direct email to 605 English households related to people living with SMA / bereaved by SMA; our, other SMA charities’, and the campaign group, TreatSMA’s, social media channels.

We received:

• 128 returns describing the health-related impacts of SMA for 128 people living with SMA Types 1-3. 61% from the adult / young person, 51% from the main unpaid carer. 52% were about people with SMA age 0 – 17 years; 48% about those age 18+ years.
• returns describing the health-related impacts of SMA for 3 people living with SMA Type 4
• 5 returns from people bereaved by SMA

We also sought people’s views on the impact of SMA on their day to day lives and the treatment nusinersen. We heard from: 56 with the condition; 55 main unpaid carers; 21 other relatives; 5 bereaved by SMA; 26 parents of children treated with nusinersen.

In the same way, we sought the experiences of parents whose children had been treated by nusinersen. We received 22 replies and added to this replies from our recent Scottish survey – 4 from England, 3 from Scotland.

Based on the prevalence of SMA of 1 – 2 in every 100,000, we estimate we have gathered the experiences of some 14-28 % of those diagnosed with the condition in England.

Summaries of the survey results were submitted to NICE as Appendices to our submission and were as follows:

Appendix 1 Impact of SMA – all Types 1 – 3

Appendix 2 Impact of SMA Types 1, 1 / 2, 2 ages 0 – 17 years Biggest Challenges (comments)

Appendix 3 Impact of SMA Types 1, 1 / 2, 2 ages 18 + years Biggest Challenges (comments)

Appendix 4 Impact of SMA Types 2 / 3, 3 ages 0 – 17 years Biggest Challenges (comments)

Appendix 5 Impact of SMA Types 2 / 3, 3 ages 18+ years Biggest Challenges (comments)

Appendix 6 Parents / carers of those treated with nusinersen – impact and expectations

Appendix 7 Parents / carers of those treated with nusinersen – impact and expectations (comments)

Appendix 8 Summary of all views about access to nusinersen

Appendix 9 Comments about priority suggestions / other comments for NICE

Appendix 10 Impact and Views Type 4 summary