Grant Support Available to Families on Lower Incomes in England

28 August 2020 / Posted in: Information, Support

National charity Family Fund, received £10m from the Department for Education to provide extra grants. The grants can be used to buy computers and play equipment as well as household items such as cookers and fridges.

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NICE Widens Scope of its Appraisal of Zolgensma

28 August 2020 / Posted in: Treatments & Research

This will now be in line with the European marketing authorisation and will now also include those who have 5qSMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene and, if evidence allows, consideration may be given to a sub-group of people who are pre-symptomatic. This widens the possibilities and is what patient groups have been advocating.

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Nice Announces Next Stages in Appraisal of Risdiplam / Evrysdi

28 August 2020 / Posted in: Treatments & Research

When we heard in April this year that NICE’s risdiplam appraisal was to be put on hold due to Covid-19, patient groups wrote jointly to NICE urging them to reinstate the process at the earliest possible time due to the great potential this treatment has for the SMA Community. We were pleased to hear this week that the appraisal is again progressing.

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Campaign for Step-Free Access at all Train Stations

24 August 2020 / Posted in: Information, Support

The Step-Free Access Campaign, set up by Ian Cook, is about petitioning for step-free access at all railway stations, and on trains, as so many are still inaccessible for disabled people. Add your voice to the campaign.

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About the Young Scientists who Make Up the UK SMA Research Conference’s Committee

24 August 2020 / Posted in: Treatments & Research

Six dedicated young PhD students from institutions across the UK are organising an online SMA research conference on 10th & 11th September. Here, you can find out more about them, their work and their interests outside the laboratory!

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FAQs About Roche’s Risdiplam Compassionate Use Programme (CUP) for Those who have SMA Type 1 or 2

21 August 2020 / Posted in: Treatments & Research

These cover who may be eligible for access, the application process and how the treatment programme will work. They also cover some other questions that have been asked about what future access routes to risdiplam there may be for those who have Type 3.

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Biogen: Community Update on European / Global Access to Nusinersen

20 August 2020 / Posted in: Treatments & Research

Biogen has provided the latest update on access to nusinersen treatment around the world. There are now 29 European countries that have access to nusinersen via regular reimbursement.

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Back to School – Webinar & Online Q&A

18 August 2020 / Posted in: Information, Support

Many parents of children with serious health needs have concerns and unanswered questions about their children returning to school in September. WellChild have organised an online Q&A, on 20th August, with a panel of experts from Department of Education, law, health and education to help answer some of these burning questions.

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EMA Validates Roche's Marketing Authorization Application for Risdiplam

17 August 2020 / Posted in: Treatments & Research

This begins the formal scientific evaluation process by EMA’s CHMP. It gives the green light for the compassionate use eligibility criteria in the UK to include patients who have SMA Type 1 and Type 2 who have exhausted all therapeutic options, or patients who have SMA Type 1 and Type 2 who have had their treatment interrupted as a direct result of the COVID-19 pandemic.

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Motability - One Big Day Online

14 August 2020 / Posted in: Information, Support

Motability's 2020 One Big Day events have had to be cancelled due to Covid-19. However, on 10th September, they are hosting a One Big Day Online event through their Facebook page.

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