Latest News: Treatments & Research

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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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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Roche’s Risdiplam Meets Primary Endpoint in SUNFISH Trial in People with SMA Type 2 or 3 SMA

12 November 2019 / Posted in: Treatments & Research

Risdiplam (previously known as RG7916) is an orally-administered, small molecule that encourages the “back-up gene”, SMN2, to produce more of the functional SMN protein.

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SMA Europe Awards Four New Grants to Cutting-Edge Research Projects

08 November 2019 / Posted in: Treatments & Research

Based on the recommendations received by its Scientific Advisory Board, SMA Europe has awarded a total of 544,900 Euros to four cutting-edge SMA research projects through its 10th Call for Research Proposals.

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New Study Improves Understanding of Impaired SMA Pathway

08 November 2019 / Posted in: Treatments & Research

Targeting the protein chondrolectin may provide a beneficial accessory therapy for SMA, by helping to stabilise and strengthen the connection of lower motor neurons to their muscles. Read more in this article from our Scientific Research Correspondent, Dr James Sleigh.

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The Intrathecal AVXS-101 Clinical Trials For SMA Have Been Put On Hold In The USA

30 October 2019 / Posted in: Treatments & Research

On 30th October, Novartis announced that the FDA in the US has placed a partial hold on clinical trials of intrathecal (lumbar puncture) injections of AVXS-101 for SMA patients. This development does not affect the intravenous (IV) administration of Zolgensma, also known as AVXS-101.

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Action For Access

25 October 2019 / Posted in: Information, Treatments & Research, Support

Genetic Alliance UK have launched this campaign to improve access to potentially life-changing treatments for people affected by rare diseases (including SMA). We're backing their campaign and calling for #ActionForAccess. Find out how you can get involved too.

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Biogen Communicates Updated NURTURE Study Results for the European Community

24 October 2019 / Posted in: Treatments & Research

The following updated results were formally presented at the Cure SMA Annual SMA Conference in Anaheim, California and the 5th Congress of the European Academy of Neurology (EAN) in Oslo, Norway.

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SMA Europe Report from Warsaw, Poland

24 October 2019 / Posted in: Treatments & Research

Polish member organisation, Fundacja SMA, were excellent hosts for SMA Europe’s biannual meeting between 10th & 12th October. The General Assembly is made-up of Patient Organisations from 19 member countries with up to two delegates from each (for SMA UK - Liz Ryburn, Support Services Manager, and Hassan Sobati, Trustee).

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Two new studies show positive effects of nusinersen in later-onset SMA

24 October 2019 / Posted in: Treatments & Research

Two small, observational studies of the effects of nusinersen in patients with later-onset SMA have recently been published, both of which describe clinically meaningful improvements caused by the treatment.

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