Latest News: Treatments & Research

Nusinersen Managed Access Agreement – Update on Progress with Services for Adults

02 December 2019 / Posted in: Treatments & Research

NHS England has let the Patient Advocacy Groups (PAGs) know which hospitals now have contracts to offer adults treatment, and their plans to address any service gaps.

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FDA Grants Priority Review to Risdiplam for the Treatment of SMA

25 November 2019 / Posted in: Treatments & Research

The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA.

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Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of SMA

25 November 2019 / Posted in: Treatments & Research

SRK-015 is an experimental therapy directed at the muscle, that aims to reverse or restrict the atrophy and weakness experienced by SMA patients.

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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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SMA UK Presenting Today At TREAT-NMD's SMA Masterclass

30 October 2019 / Posted in: Treatments & Research

Liz and Jackie, from SMA UK’s Support Services Team, are presenting today at TREAT-NMD’s SMA Masterclass on patient involvement in therapy development.

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