24 September 2018 / Posted in: Treatments & Research
NICE have announced that the public registration facility to observe the next committee meeting about nusinersen on 23rd October will open on 25th September 2018 on their website.
21 September 2018 / Posted in: Information, Support
The charity Contact for Families of Disabled Children say that they have been inundated with calls about issues surrounding school transport. There are a number of support resources available about this on their website, along with an upcoming live Facebook Q&A, and Midlands workshops.
19 September 2018 / Posted in: Help Us
We can’t wait for this year’s Christmas Jumper Day for SMA on Friday 7th December and we hope you can join us to make this one the biggest yet!
19 September 2018 / Posted in: Treatments & Research
Patient groups and clinicians have been invited to take part in this consultation which closes on 17th October. Treatment topics usually reach what is called ‘the scoping stage’ around the time the company plan to apply for a licence for its drug in the UK or Europe. SMA UK will be submitting a response.
11 September 2018 / Posted in: Treatments & Research
Thanks to those who responded to our survey asking for your views on newborn screening. We concluded that a further urgent review of a newborn screening programme for 5q SMA in the UK is imperative.
08 September 2018 / Posted in: Treatments & Research
On 5th September we submitted our response to, NICE’s consultation paper which had been released on 14th August with the devastating news that they had not recommended nusinersen for funding by the NHS. You can read our response here.
31 August 2018 / Posted in: Information, Support
It's back to school time which can mean higher than normal costs, with new uniforms, stationery, bags etc. You might want to check out Contact's 'Money Matters' guide which includes details on the financial help you're entitled to if your child's at school.
29 August 2018 / Posted in: Treatments & Research
This is so that, as a matter of urgency, an MAA that merges seamlessly with the current EAP is set up and ensures that all others are also able to access nusinersen at the earliest possible opportunity.
24 August 2018 / Posted in: Treatments & Research
This update recently went out to those on our mailing list. It includes details on the MDUK parliamentary drop-in session that's been organised for 11th September, how you can contact your MP, responding to NICE by 5th Sept, and media involvement.
Biogen Announces November Closure of the Expanded Access Programme for Infants Newly Diagnosed with SMA Type 1
23 August 2018 / Posted in: Treatments & Research
We urged Biogen not to take this step which creates yet more immense distress and worry for families. We will continue to focus our advocacy for access to treatment on the urgent need for NICE, NHS England and Biogen to agree the terms of a managed access agreement to replace this EAP ahead of 1 November, as well as the need for a long-term sustainable plan for access.