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Latest News: Treatments & Research

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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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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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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Campaign to Protect ERNs - Add Your Voice

08 October 2018 / Posted in: Treatments & Research

Genetic Alliance UK has launched a campaign to ensure the UK's continued participation in European Reference Networks (ERNs) following our exit from the European Union.

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AveXis Clinical Trial for Infants Aged Less than Six Months Old with SMA Type 1 in the UK

27 September 2018 / Posted in: Treatments & Research

This Phase 3 study called STR1VE-EU, will evaluate the safety and efficacy of a one-time intravenous infusion of AVXS-101 in approximately 30 European infants.

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Public Registration: NICE Technology Appraisal Advisory Committee Meeting

24 September 2018 / Posted in: Treatments & Research

NICE have announced that the public registration facility to observe the next committee meeting about nusinersen on 23rd October will open on 25th September 2018 on their website.

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NICE Opens Draft Scoping Consultation for 'Breakthrough' AveXis Gene Therapy for SMA Type 1

19 September 2018 / Posted in: Treatments & Research

Patient groups and clinicians have been invited to take part in this consultation which closes on 17th October. Treatment topics usually reach what is called ‘the scoping stage’ around the time the company plan to apply for a licence for its drug in the UK or Europe. SMA UK will be submitting a response.

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Our Response to the UK National Screening Committee Review of Screening for SMA

11 September 2018 / Posted in: Treatments & Research

Thanks to those who responded to our survey asking for your views on newborn screening. We concluded that a further urgent review of a newborn screening programme for 5q SMA in the UK is imperative.

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Our Response to NICE's Consultation Paper

08 September 2018 / Posted in: Treatments & Research

On 5th September we submitted our response to, NICE’s consultation paper which had been released on 14th August with the devastating news that they had not recommended nusinersen for funding by the NHS. You can read our response here.

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