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Latest News: Treatments & Research

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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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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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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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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Biogen wins UK Prix Galien award for nusinersen, but we are all still waiting for news from NICE

23 November 2018 / Posted in: Treatments & Research

On 15th November, Biogen was announced as the winner of the Orphan Product Award for Spinraza® (nusinersen). They comment, ‘we must see this translated into availability for the patients who desperately need it’. We agree and will keep working on this.

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Rare Disease Day 2019 Theme Announced

19 November 2018 / Posted in: Treatments & Research

The theme for the day has been announced as ‘Bridging Health and Social Care’. The 12th edition of the campaign will focus on the need to bridge the gaps in the coordination between medical, social and support services for people affected by a rare disease.

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SMA UK Busy Heading Out and About

13 November 2018 / Posted in: Treatments & Research

Last week, along with MDUK, we were patient reps at NICE’s scoping meeting for AVXS-101 for SMA Type 1. We also attended Genetic Alliance UK’s workshop for rare disease groups discussing a Patient Charter for Newborn Screening in the UK.

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