Charities jointly write to NICE to ensure Nusinersen is on next committee meeting's agenda

04 December 2018 / Posted in: Treatments & Research

Today, SMA UK, MDUK and TreatSMA urged NICE to draw the appraisal process to a satisfactory conclusion at their next available meeting on 6th February 2019. Read the letter here.

Read full story

Scholar Rock announce SRK-015 orphan drug designation news and publish pre-clinical data

04 December 2018 / Posted in: Treatments & Research

Pharmaceutical company Scholar Rock has announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted a positive opinion on their experimental drug for SMA, SRK-015.

Read full story

Newlife #FixTheFour Campaign

29 November 2018 / Posted in: Information, Support

Newlife are calling on the government to help those children whose lives are at risk because they don’t have the specialist equipment they need. Their report, 'Equipment Crisis for Disabled Children', outlines a four-point-plan to improve equipment services for disabled children and their families.

Read full story

SMA UK, MDUK and TreatSMA send joint letters to NICE, Biogen and NHS England

29 November 2018 / Posted in: Treatments & Research

As an act of ‘good faith’ in their commitment to progress discussions to a satisfactory conclusion, we urge all three parties to agree with immediate effect to implement, a ‘bridging solution’ for access to nusinersen for any infants with SMA Type 1.

Read full story

SMA UK attended APPG Psychological Support Inquiry Launch

28 November 2018 / Posted in: Information, Support

On Wednesday 21st November, we joined MDUK at Parliament for the launch of this inquiry, which included discussing ways to improve the availability of appropriate psychological support. You can read their full APPG report here.

Read full story

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