Latest News: Treatments & Research

Access to Zolgensma Submission Sent to the Scottish Medicines Consortium

09 December 2020 / Posted in: Treatments & Research

SMA UK and MDUK have made a joint submission, which you can read here, in which we strongly support access to this treatment. We expect the Committee to meet and publish their decision in the first quarter of 2021.

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Action to Address Lack of Access to the Risdiplam Early Access to Medicines Scheme for Adults in the East of England

04 December 2020 / Posted in: Treatments & Research

We are working through various routes to urge the Trust to change its position and, in the meantime, to address how people living in this area of the country who meet the EAMS eligibility criteria can get access to the risdiplam EAMS.

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New Articles on Aspects of Clinical Care Research from our Correspondent

25 November 2020 / Posted in: Treatments & Research

Dr. Alex Murphy has put together these two articles - the first around exercise and SMA, the second discussing a study assessing the use of a physical function assessment tool.

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SMA UK Sends Evidence to Biogen for the NICE Review Considering Whether People who have SMA Type 3 and Unable to Walk can Benefit from Nusinersen Treatment

23 November 2020 / Posted in: Treatments & Research

We are very aware that this review will be focusing on clinical evidence from Biogen and the clinical community. However, we consider it is also vital that NICE hears the wider ranging evidence we have gathered.

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If you or your child live in England, have SMA Type 3 and have lost the ability to walk, please respond to this survey

20 November 2020 / Posted in: Treatments & Research

If NICE recommends that England's MAA for nusinersen should change to include you / your child, would you want to access this treatment? NHS England and the Neuromuscular Centres need to be ready for the possibility of a positive recommendation for change. SMA UK has agreed to put out this brief survey (closes 13th December) to help with any potential future planning. This does not guarantee a positive recommendation will be made.

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Out of the Shadows - Report by The Neurological Alliance

12 November 2020 / Posted in: Information, Treatments & Research, Support

This new report, launched today, is calling for better treatment and care for the 150,000 children and adults living with a rare neurological condition, including SMA, in England.

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SMA UK's and MDUK's Joint Submission to NICE as to Whether they Should Recommend that NHS England Funds Risdiplam

04 November 2020 / Posted in: Treatments & Research

Thank you to those of you who completed the survey we ran about your views on this - they were crucial for the submission we made. The first committee meeting is scheduled for 11th May 2021. You can see the submission and survey results through here.

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I:DNA Immersive Art Installation

02 November 2020 / Posted in: Information, Treatments & Research, Support

Prof Felicity Boardman has done extensive research on the social and ethical implications of genomic medicine from the perspectives of people with genetic conditions. I:DNA is a virtual, immersive art installation which is an artistic representation of all this research. You can ask questions ahead of a live Q&A on 11th November.

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NICE Announces Start of Review to Consider if People who have SMA Type 3 who're Unable to Walk can Benefit from Nusinersen Treatment

29 October 2020 / Posted in: Treatments & Research

NICE has today announced that it has begun the process to review data collected as part of the MAA for nusinersen. Specifically, it will consider whether people who have SMA Type 3 who are unable to walk can benefit from the treatment and therefore should be included in the MAA.

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Scholar Rock Announces Encouraging Interim Data from the TOPAZ Phase 2 Trial of SRK-015

28 October 2020 / Posted in: Treatments & Research

SRK-015 is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. TOPAZ is evaluating SRK-015 across three cohorts of people, who have SMA Types 2 and 3, using motor function scales to measure meaningful endpoints.

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