SMA UK Activity During the Week of Rare Disease Day

22 February 2019 / Posted in: Treatments & Research

Monday, we’re at the Parliamentary 'drop-in' session about access to nusinersen; Tuesday, our Scientific Research Correspondent will be attending Royal Holloway’s Rare Disease Day event; on Rare Disease Day itself, we’ll be at a Cambridge Lecture evening about research and have a a notice in the Guardian Rare Diseases supplement.

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UK SMA Research Day 2019

21 February 2019 / Posted in: Treatments & Research

Hosted at Keele University in Keele Hall, the latest UK SMA researcher meeting was organised and chaired by Dr. Melissa Bowerman on Wednesday 23rd January 2019. Scientists of all career stages representing many UK research institutions were in attendance for a full day of excellent presentations on SMA. Read this summary by our Scientific Research Correspondent, Dr James Sleigh.

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National programme for carrier or newborn screening for SMA not recommended by UK National Screening Committee (NSC)

18 February 2019 / Posted in: Treatments & Research

This was their conclusion following their consultation and evidence review. We will continue to work with other groups to push for both a review of the evidence criteria and a further urgent review of a newborn screening programme for 5q SMA. You can read our submission to the consultation and more about NSC's recommendation here.

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Please invite your MP to the 25th February Parliamentary ‘drop in’ about access to nusinersen (Spinraza) for England, Wales and Northern Ireland

14 February 2019 / Posted in: Treatments & Research

MDUK and SMA UK have jointly arranged this event. Parents and adults representing the SMA Community will be telling MPs about their experiences of living with SMA, and will be joined by clinicians to talk about the importance of access to nusinersen. They will be outlining the actions MPs can take to show their support and raise the matter further.

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Subject to successful sign off, nusinersen is expected to be routinely available in Scotland for those with SMA Types 1, 2 and 3 from April 2019

12 February 2019 / Posted in: Treatments & Research

Nusinersen has been routinely available to children with SMA Type 1 in Scotland since May 2018. This will continue. The SMC has now granted nusinersen ultra-orphan designation, allowing the treatment to be reviewed for those with SMA Types 2 and 3 through its new ultra-orphan appraisal process with the prospect of it being made available for at least three years while further outcome data is gathered.

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NICE announce Committee Meeting on 6th March 2019

01 February 2019 / Posted in: Treatments & Research

This will be the third meeting to review whether NICE will recommend nusinersen for funding by NHS England. This meeting will be in Manchester again. As soon as we have more details we will let you know.

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MDUK's Benefits and Grants Survey

31 January 2019 / Posted in: Information, Support

Muscular Dystrophy UK's ‘Focus on Disability Benefits’ campaign aims to ensure that people with neuromuscular conditions can access fair and informed assessments for support that meets their needs. This survey is about your experience of the application process for any benefits you receive.

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Ask Your MP to Speak in Debate on Support for Children with Life-limiting Conditions

24 January 2019 / Posted in: Information, Support

Together for Short Lives are asking people to write to their MPs to ask them to speak in this debate. The meeting will take place at 9:30am on Tuesday 29 January 2019 in Westminster Hall.

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New Campaign: Make Cervical Cancer Screening Accessible For Disabled Women

24 January 2019 / Posted in: Information, Support

Fiona Anderson, a wheelchair user who has a muscle-wasting condition, has set up a petition to ensure disabled women have equal access to cervical cancer screening - this includes by making hoists available at every NHS medical centre.

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Find out about our fundraisers Find out about our fundraisers

24 January 2019 / Posted in: Help Us

Some of our Fundraising Teams and Fund Holders have been sharing their thoughts with us on their experiences of fundraising for SMA.

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