Tel: 01789 267520

Updated Factsheet from Disability Rights UK

08 February 2018 / Posted in: Information & Support

The comprehensive factsheet will be useful for anyone who is claiming PIP (Personal Independence Payment) for the first time, or who has been in receipt of DLA and invited to apply for PIP instead.

Read full story

Our Patient Group Submission to the Scottish Medicines Consortium (SMC)

06 February 2018 / Posted in: Research

Thanks to those who responded to our invitation to take part in a survey to tell us about your experiences of SMA and your views on access to nusinersen. Read a copy of our submission and a summary of the survey results, here.

Read full story

Rare Disease Implementation Plans for England Published

01 February 2018 / Posted in: Information & Support, Research

The Department of Health and Social Care and NHS England have published their implementation plans for the UK Strategy for Rare Diseases. England is now in line with Scotland, Wales and Northern Ireland who already have plans in place.

Read full story

AveXis Announce Additional Trials of AVXS-101 for SMA

25 January 2018 / Posted in: Research

AveXis have provided an overview of their expanded program of clinical development for their gene therapy drug AVXS-101 in SMA, which incorporates two new trials to include a broader set of SMA patients.

Read full story

Biogen’s SMA Community Update Confirms the NICE Appraisal Process

22 January 2018 / Posted in: Research

It also contains a little bit more information about how the possibility of a Managed Access Programme will be explored.

Read full story

NICE Announces Appraisal Route for Nusinersen

19 January 2018 / Posted in: Research

This will be via the Single Technology Appraisal (STA) route which is not set up to assess rare disease drugs. However, there is some promising news which may see an interim Managed Access Agreement (MAA) put in place which could give more people with SMA access.

Read full story

New 2018 Events New 2018 Events

12 January 2018 / Posted in: Help Us

Fancy a new challenge for 2018? Or holding a community event to raise vital SMA awareness? Check out our events!

Read full story

Fundraising Support Group Fundraising Support Group

12 January 2018 / Posted in: Help Us

Would you like to help SMA Support UK raise funds and awareness to ensure our vital services continue? If so, then why not consider joining our Midlands Fundraising Support Group!

Read full story

Transition to Adult Services

11 January 2018 / Posted in: Information & Support

Together for Short Lives has produced this factsheet to help parents think about the different elements of transition that need to be planned for and to provide tips to help the process feel as smooth as possible.

Read full story

The Updated Standards of Care Have Now Been Published

08 January 2018 / Posted in: Research

This means work can now start on writing the updated Family Guide. SMA Support UK will be working with others on this. In the meantime, you can read a summary of the ENMC workshop review of the standards.

Read full story