#VoteSEND - Make Disabled Children a Priority in the General Election

20 November 2019 / Posted in: Information, Support

There are 1.1 million disabled children in the UK, but the system designed to support them and their families is failing. The Disabled Children's Partnership want to hear from all parties and candidates, as we approach the general election, as to what they will do to mend the system. Find out how you can get involved.

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World Children's Day - Jane Velkovski

19 November 2019 / Posted in: Information, Support

11-year-old Jane Velkovski from North Macedonia, who has SMA, will be joining UNICEF and speaking at a global summit at the United Nations Headquarters in New York. Children and young people will be there leading a call to action to protect and promote child rights.

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Changes to Deadlines and Timescales of CHC Appeals Processes

15 November 2019 / Posted in: Information, Support

It has been confirmed that CCGs are now able to set the time that individuals have to challenge an eligibility decision for NHS Continuing Healthcare, leading to a 'postcode lottery'.

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Survey About What Progress There Has Been With Children's Access To Nusinersen In The UK

14 November 2019 / Posted in: Information, Treatments & Research, Support

SMA UK, MDUK and TreatSMA have launched this survey for the paediatric SMA Community to gather information about people’s experiences of trying to access nusinersen (Spinraza). Closing date is Sunday 1st December. An adult survey will follow in due course.

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Roche’s Risdiplam Meets Primary Endpoint in SUNFISH Trial in People with SMA Type 2 or 3 SMA

12 November 2019 / Posted in: Treatments & Research

Risdiplam (previously known as RG7916) is an orally-administered, small molecule that encourages the “back-up gene”, SMN2, to produce more of the functional SMN protein.

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SMA Europe Awards Four New Grants to Cutting-Edge Research Projects

08 November 2019 / Posted in: Treatments & Research

Based on the recommendations received by its Scientific Advisory Board, SMA Europe has awarded a total of 544,900 Euros to four cutting-edge SMA research projects through its 10th Call for Research Proposals.

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New Study Improves Understanding of Impaired SMA Pathway

08 November 2019 / Posted in: Treatments & Research

Targeting the protein chondrolectin may provide a beneficial accessory therapy for SMA, by helping to stabilise and strengthen the connection of lower motor neurons to their muscles. Read more in this article from our Scientific Research Correspondent, Dr James Sleigh.

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New Survey: Your Views On Living With A Rare Disease

30 October 2019 / Posted in: Information, Support

The Minister for rare disease at the Department of Health and Social Care, Baroness Blackwood, has announced a national conversation to understand how we can better care for people who are living with a rare disease. Survey closes 29th November.

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The Intrathecal AVXS-101 Clinical Trials For SMA Have Been Put On Hold In The USA

30 October 2019 / Posted in: Treatments & Research

On 30th October, Novartis announced that the FDA in the US has placed a partial hold on clinical trials of intrathecal (lumbar puncture) injections of AVXS-101 for SMA patients. This development does not affect the intravenous (IV) administration of Zolgensma, also known as AVXS-101.

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Action For Access

25 October 2019 / Posted in: Information, Treatments & Research, Support

Genetic Alliance UK have launched this campaign to improve access to potentially life-changing treatments for people affected by rare diseases (including SMA). We're backing their campaign and calling for #ActionForAccess. Find out how you can get involved too.

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