SMA UK Calling for Urgent Access to Nusinersen

04 March 2019 / Posted in: Treatments & Research

On Wednesday 6th March, we will be at NICE’s third committee meeting working hard to express the urgent need for a positive recommendation.

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'Time is Running Out' - Please send this powerful handout and template letter to your MP today

26 February 2019 / Posted in: Treatments & Research

A huge thank you to all who personally gave copies of these and spoke about their lives to MPs at yesterday's Access to nusinersen parliamentary drop-in session. You can contact your MP, asking them to use these to write to NICE, Biogen and NHS England

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Join Ride Scorpion for SMA Join Ride Scorpion for SMA

25 February 2019 / Posted in: Help Us

Join us on 18th May to cycle the Cotswolds and raise vital funds to support people living with Spinal Muscular Atrophy.

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Marathon in May for SMA is back! Marathon in May for SMA is back!

24 February 2019 / Posted in: Help Us

Our virtual event – Marathon in May for SMA – is back and we hope that as many of you as possible will be able to take part.

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Your help needed to find health professionals to take part in study into costs and treatment of SMA

22 February 2019 / Posted in: Treatments & Research

A biotechnology company is investigating a new treatment for SMA and wants to arrange interviews with health visitors, community nurses, physios, OTs, nurses in neurology wards, nutritionists, neurologists and pulmonologists who have seen a minimum of 2 patients with SMA Type 1, 2 or 3 in the last 12 months. Please pass this request on to the health professionals who support you to see if they wish to get involved.

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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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