$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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Consultation About Spinal Muscular Atrophy UK's Priorities

09 October 2018 / Posted in: Information, Treatments & Research, Support

We are now starting to work out priorities for the new organisation, including how and when the funds we hope to raise should be spent. We are very keen to hear the views of as many people in the SMA Community as possible. The survey closes on 11th November.

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Biogen’s ‘Biomarkers’ Research to Find Ways to Tell Whether a Medication Has Been Successful

09 October 2018 / Posted in: Treatments & Research

At the 23rd International Annual Congress of the World Muscle Society, pharmaceutical company Biogen presented the findings of blood tests done on over 300 participants receiving nusinersen in their clinical trials.

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New Results from Biogen’s NURTURE Study of Pre-Symptomatic Infants with SMA

09 October 2018 / Posted in: Treatments & Research

At the 23rd International Annual Congress of the World Muscle Society, pharmaceutical company Biogen announced its latest findings.

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Leonard Cheshire's Change 100 Programme Now Open For Applications

08 October 2018 / Posted in: Information, Support

The Change 100 scheme was launched in 2014 to enable young disabled people to obtain useful work experience and mentoring. Applications are now open until 16th January 2019.

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