SMA UK's and MDUK's Joint Submission to NICE as to Whether they Should Recommend that NHS England Funds Risdiplam

04 November 2020 / Posted in: Treatments & Research

Thank you to those of you who completed the survey we ran about your views on this - they were crucial for the submission we made. The first committee meeting is scheduled for 11th May 2021. You can see the submission and survey results through here.

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I:DNA Immersive Art Installation

02 November 2020 / Posted in: Information, Treatments & Research, Support

Prof Felicity Boardman has done extensive research on the social and ethical implications of genomic medicine from the perspectives of people with genetic conditions. I:DNA is a virtual, immersive art installation which is an artistic representation of all this research. You can ask questions ahead of a live Q&A on 11th November.

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NICE Announces Start of Review to Consider if People who have SMA Type 3 who're Unable to Walk can Benefit from Nusinersen Treatment

29 October 2020 / Posted in: Treatments & Research

NICE has today announced that it has begun the process to review data collected as part of the MAA for nusinersen. Specifically, it will consider whether people who have SMA Type 3 who are unable to walk can benefit from the treatment and therefore should be included in the MAA.

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Beacon: How Continuing Healthcare Will Resume Post-COVID 19

29 October 2020 / Posted in: Information, Support

In August 2020, new government guidance set out how NHS Continuing Healthcare (CHC) would be reintroduced in England from 1st September 2020, along with a new Hospital Discharge Policy. Beacon have provided further information around this.

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Scholar Rock Announces Encouraging Interim Data from the TOPAZ Phase 2 Trial of SRK-015

28 October 2020 / Posted in: Treatments & Research

SRK-015 is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. TOPAZ is evaluating SRK-015 across three cohorts of people, who have SMA Types 2 and 3, using motor function scales to measure meaningful endpoints.

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Write to your MP: Please Increase Legacy Benefits

27 October 2020 / Posted in: Information, Support

It’s been 6 months since DWP increased Universal Credit by £20 a week but failed to give the same increase to those on legacy benefits. Now is our opportunity to make sure the Chancellor doesn’t let over two million people go without the £20 per week increase for another year. Write to your MP.

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Cell and Gene Therapy Engagement Workshops

23 October 2020 / Posted in: Information, Treatments & Research, Support

Genetic Alliance UK are organising a series of virtual workshops on the topic of cell and gene therapies, sometimes referred to as Advanced Therapy Medicinal Products (ATMPs). Date to register closes 26th October.

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Community Connections: 'PARENTS! Ask Us Anything'

23 October 2020 / Posted in: Information, Support

If you're a parent of a child, teenager or young adult with SMA and there’s something that you’ve wanted to know but you’ve been too anxious, or you’ve just never had the opportunity, to ask a young adult with SMA, this is your chance! Jordanne and Josh are here to answer your questions. Please send them in by 31st October.

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Survey About the Treatment of Childhood Neurological Conditions

23 October 2020 / Posted in: Information, Support

The British Paediatric Neurology Association and James Lind Alliance have just launched a Priority Setting Partnership survey to help define the Top 10 unanswered questions about the treatment of Childhood Neurological Conditions. Find out more and take the survey (closes Jan 2021).

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Kidz to Adults - From Venue Floor to Virtual Tour!

12 October 2020 / Posted in: Information, Support

As for the first time in almost 20 years Kidz to Adultz are unable to be on the exhibition floor, they've launched: Venue to Virtual (9th-13th November). The online platform offers visitors free virtual access to many exhibitors, companies, voluntary and support organisations.

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