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AVXS-101 to receive expedited regulatory review in the US and Europe

06 December 2018 / Posted in: Treatments & Research

Novartis has announced that the US Food and Drug Administration (FDA) has accepted for review with Priority Status their new drug application for the experimental gene therapy for SMA, AVXS-101, which has now been renamed ZOLGENSMA.

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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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Our virtual Christmas Tree is now live Our virtual Christmas Tree is now live

05 December 2018 / Posted in: Help Us

Choose a bauble to hang on our SMA UK tree and leave a festive message

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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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Coffee and Cake Day Coffee and Cake Day

04 December 2018 / Posted in: Help Us

Why not hold a festive bake sale in December to get family, friends and neighbours together over Christmas!

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

30 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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