Latest News: Treatments & Research
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.
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.
21 August 2018 / Posted in: Treatments & Research
Clinicians and SMA Charities yesterday expressed the need for urgent action. You can read the letter sent to NICE, NHS England and the National Institute for Health Research, here.
15 August 2018 / Posted in: Treatments & Research
Biogen has provided this community statement in response to yesterday's announcement from NICE.
13 August 2018 / Posted in: Treatments & Research
Nusinersen not recommended for funding by NICE is the devastating decision for so many, announced today. One glimmer of hope, however, is that it does leave the door open for a Managed Access Agreement. Comments are needed by 5th September.
03 August 2018 / Posted in: Treatments & Research
The National Screening Committee (NSC) has invited us to give comments and feedback on its June 2018 evidence review. Their current recommendation is ‘systematic population screening programme not recommended’. When we submit our response, we would like to represent views from the SMA community about screening newborns for SMA. You can let us know your thoughts and take our survey.
02 August 2018 / Posted in: Treatments & Research
We have received this community update which includes Q and As about the potential risk of hydrocephalus.