Tel: 01789 267520

Latest News: Treatments & Research

AveXis Clinical Trial for Infants Aged Less than Six Months Old with SMA Type 1 in the UK

27 September 2018 / Posted in: Treatments & Research

This Phase 3 study called STR1VE-EU, will evaluate the safety and efficacy of a one-time intravenous infusion of AVXS-101 in approximately 30 European infants.

Read full story

Public Registration: NICE Technology Appraisal Advisory Committee Meeting

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.

Read full story

NICE Opens Draft Scoping Consultation for 'Breakthrough' AveXis Gene Therapy for SMA Type 1

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.

Read full story

Our Response to the UK National Screening Committee Review of Screening for SMA

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.

Read full story

Our Response to NICE's Consultation Paper

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.

Read full story

Charities write to Biogen urging them and NHS England to be Flexible on Price

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.

Read full story

Access to Nusinersen Update & How You Can Help

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.

Read full story

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.

Read full story

Clinicians and SMA Charities Protest NICE Decision

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.

Read full story

Biogen’s Update to the SMA Community Following Publication of NICE’s Consultation Paper

15 August 2018 / Posted in: Treatments & Research

Biogen has provided this community statement in response to yesterday's announcement from NICE.

Read full story