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Autumn/Winter Events Feedback

24 October 2018 / Posted in: Support

If you have attended one of our Autumn/Winter Social Events so far we hope you had an enjoyable day. We would like your feedback and have put together a short survey to gather your views.

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Campaign for the Government to put Children at the Heart of its Spending Plans

23 October 2018 / Posted in: Information

We support this campaign which shows there is compelling evidence that the services and support that children and young people rely on are at breaking point.

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Project Move

22 October 2018 / Posted in: Information

Project Move is an exciting joint venture between Alder Hey and The University of Liverpool looking at redesigning and modernising children’s powered wheelchairs. Find out how you could get involved by attending their event.

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AveXis Community Update on Submitting their Application for AVXS-101

19 October 2018 / Posted in: Treatments & Research

AveXis yesterday submitted their regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1. This is an important initial step toward the potential approval of AVXS-101, a gene replacement therapy candidate for the treatment of the underlying root cause of SMA.

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$3 million 'Breakthrough' prize for Developers Behind Spinraza

18 October 2018 / Posted in: Treatments & Research

An article in The Guardian, published yesterday, describes how C Frank Bennett, senior vice president of research at Ionis Pharmaceuticals, and Adrian Krainer of Cold Spring Harbor Laboratory, have won the prize for developing Spinraza.

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SMA UK’s submission to NICE’s scoping consultation on AVXS-101 treatment for infants with SMA Type 1

17 October 2018 / Posted in: Treatments & Research

England’s regulatory body, NICE, has invited a wide range of clinical and patient groups to comment on this treatment. You can read what we have said to NICE here.

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The Big Give Christmas Challenge 2018 The Big Give Christmas Challenge 2018

16 October 2018 / Posted in: Help Us

Join us for The Big Give Christmas Challenge 2018 and double your donation!

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Roche Announces Some Results from their Firefish and Sunfish Trials

09 October 2018 / Posted in: Treatments & Research

On 3rd October at the 23rd International Annual Congress of the World Muscle Society, Roche presented some of the results of their clinical trials of the treatment risdiplam.

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Biogen Response to the Charities' August 28th Joint Letter

09 October 2018 / Posted in: Treatments & Research

In our letter, we urged both Biogen and NHS England to be as flexible as possible in their discussion on the price of nusinersen. Read their reply here.

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Scottish Government Announces New Pathway Opens for Ultra-Orphan Treatments

09 October 2018 / Posted in: Treatments & Research

Yesterday’s announcement gives hope that nusinersen could soon be reassessed for access for those with SMA Type 2 or 3 in Scotland.

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