UK SMA Research Day 2019

06 December 2018 / Posted in: Treatments & Research

The UK SMA Research Consortium and SMA UK are inviting all SMA Researchers to the next UK SMA Research Day, which will be held January 23rd 2019, from 9am to 5pm, in the Keele Hall Salvin Room at Keele University.

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Biogen awarded the 2018 International Prix Galien as Best Biotechnology Product for Spinraza (nusinersen)

06 December 2018 / Posted in: Treatments & Research

While we still await a decision from NICE, this marks the seventh Prix Galien for Spinraza, following country recognitions in the U.S., Germany, Italy, Belgium-Luxembourg, the Netherlands and the U.K.

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Have you visited a genetic service within the NHS? Could you share your opinions for research?

05 December 2018 / Posted in: Treatments & Research

Researchers are conducting a study to explore people’s views about how patient data could be used as part of future genomic medicine services in the NHS.

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New Clinical Care Research Article: SMA Masterclass

05 December 2018 / Posted in: Treatments & Research

On the 19th of November, the first spinal muscular atrophy (SMA) masterclass took place in Rome organised by the neuromuscular network TREAT NMD. Our Clinical Care Research Correspondent, Dr Alex Murphy, has written an article summarising the topics covered within the masterclass.

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Charities jointly write to NICE to ensure Nusinersen is on next committee meeting's agenda

04 December 2018 / Posted in: Treatments & Research

Today, SMA UK, MDUK and TreatSMA urged NICE to draw the appraisal process to a satisfactory conclusion at their next available meeting on 6th February 2019. Read the letter here.

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Scholar Rock announce SRK-015 orphan drug designation news and publish pre-clinical data

04 December 2018 / Posted in: Treatments & Research

Pharmaceutical company Scholar Rock has announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted a positive opinion on their experimental drug for SMA, SRK-015.

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Newlife #FixTheFour Campaign

29 November 2018 / Posted in: Information, Support

Newlife are calling on the government to help those children whose lives are at risk because they don’t have the specialist equipment they need. Their report, 'Equipment Crisis for Disabled Children', outlines a four-point-plan to improve equipment services for disabled children and their families.

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SMA UK, MDUK and TreatSMA send joint letters to NICE, Biogen and NHS England

29 November 2018 / Posted in: Treatments & Research

As an act of ‘good faith’ in their commitment to progress discussions to a satisfactory conclusion, we urge all three parties to agree with immediate effect to implement, a ‘bridging solution’ for access to nusinersen for any infants with SMA Type 1.

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SMA UK attended APPG Psychological Support Inquiry Launch

28 November 2018 / Posted in: Information, Support

On Wednesday 21st November, we joined MDUK at Parliament for the launch of this inquiry, which included discussing ways to improve the availability of appropriate psychological support. You can read their full APPG report here.

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UK SMA Patient Registry Update

28 November 2018 / Posted in: Treatments & Research

The UK SMA Patient Registry collects clinical and genetic information from individuals living with SMA to provide an aid to clinical study recruitment and an invaluable resource to the research community. To find out more about the registry and a snapshot of the data held, please click here.

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