Support the #NeuroPledge2019

03 December 2019 / Posted in: Information, Support

The Neurological Alliance are asking parliamentary candidates to take the #NeuroPledge2019. It commits them to campaigning for improvements for people with neurological conditions, if they become an MP. Find out how you can get involved and write to your prospective MP.

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Nusinersen Managed Access Agreement – Update on Progress with Services for Adults

02 December 2019 / Posted in: Treatments & Research

NHS England has let the Patient Advocacy Groups (PAGs) know which hospitals now have contracts to offer adults treatment, and their plans to address any service gaps.

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CONCORD Survey about the Coordination of Care for Rare Conditions

28 November 2019 / Posted in: Information, Support

The COordiNated Care Of Rare Diseases (CONCORD) research study is currently looking for patients, carers and healthcare professionals to share their priorities and views about the coordination of care for rare conditions, via their online survey. Survey closes Sunday 8th December.

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FDA Grants Priority Review to Risdiplam for the Treatment of SMA

25 November 2019 / Posted in: Treatments & Research

The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA.

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Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of SMA

25 November 2019 / Posted in: Treatments & Research

SRK-015 is an experimental therapy directed at the muscle, that aims to reverse or restrict the atrophy and weakness experienced by SMA patients.

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SMA UK’s Trust Board makes Research Strategy and Funding decisions

21 November 2019 / Posted in: Treatments & Research

We are pleased to announce our SMA PhD Partnership with MDUK that is open for applications now along with our pledge to support SMA Europe’s research call. For more details and information about this and other 2019/20 grants, please click here.

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#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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