UK SMA Patient Registry Update

28 November 2018 / Posted in: Treatments & Research

The UK SMA Patient Registry collects clinical and genetic information from individuals living with SMA to provide an aid to clinical study recruitment and an invaluable resource to the research community. To find out more about the registry and a snapshot of the data held, please click here.

Read full story

Able Move Chosen as Winner of Stelios Award for Disabled Entrepreneurs 2018

26 November 2018 / Posted in: Information, Support

Able Move, a start-up company that has created a portable solution for disabled travellers, has been chosen as winner of this award, in partnership with Leonard Cheshire. The company was founded by Josh Wintersgill, a cyber-security manager from Bristol, who has SMA.

Read full story

Biogen wins UK Prix Galien award for nusinersen, but we are all still waiting for news from NICE

23 November 2018 / Posted in: Treatments & Research

On 15th November, Biogen was announced as the winner of the Orphan Product Award for Spinraza® (nusinersen). They comment, ‘we must see this translated into availability for the patients who desperately need it’. We agree and will keep working on this.

Read full story

ORR Improving Assisted Travel Consultation

19 November 2018 / Posted in: Information, Support

A consultation being run by the Office of Rail and Road (ORR) sets out proposals to change the Disabled People's Protection Policy (DPPP) Guidance for train and station operators on how to write their policies for helping disabled people to travel by rail.

Read full story

Rare Disease Day 2019 Theme Announced

19 November 2018 / Posted in: Treatments & Research

The theme for the day has been announced as ‘Bridging Health and Social Care’. The 12th edition of the campaign will focus on the need to bridge the gaps in the coordination between medical, social and support services for people affected by a rare disease.

Read full story

SMA UK Busy Heading Out and About

13 November 2018 / Posted in: Treatments & Research

Last week, along with MDUK, we were patient reps at NICE’s scoping meeting for AVXS-101 for SMA Type 1. We also attended Genetic Alliance UK’s workshop for rare disease groups discussing a Patient Charter for Newborn Screening in the UK.

Read full story

Latest NICE Community Update re Nusinersen

12 November 2018 / Posted in: Treatments & Research

This latest SMA Community update from NICE doesn't give a decision yet but states that talks are still progressing.

Read full story

Universal Credit and Universal Support

01 November 2018 / Posted in: Information, Support

The charity Turn2Us have written an article explaining how people claiming Universal Credit can get help through Universal Support which enables them to make a claim and manage ongoing payments.

Read full story

Biogen Statement to SMA Community re: EAP for SMA Type 1 in England, Wales and Northern Ireland

01 November 2018 / Posted in: Treatments & Research

Despite our urgent request, Biogen has not extended the cut off for this programme to 1st December. It ends now for any newly diagnosed infants. Read their community statement here.

Read full story

Update on Access to Nusinersen

26 October 2018 / Posted in: Treatments & Research

Thank you to the patient and clinical experts who strongly advocated for access to nusinersen at NICE’s committee meeting this week; to clinicians, families and adults who: expressed their views in their submissions to NICE; wrote letters to MPs, NICE, NHS England and Biogen; were there on the day.

Read full story