Latest News: Treatments & Research

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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Latest NICE Community Update re Nusinersen

12 November 2018 / Posted in: Treatments & Research

This latest SMA Community update from NICE doesn't give a decision yet but states that talks are still progressing.

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Biogen Statement to SMA Community re: EAP for SMA Type 1 in England, Wales and Northern Ireland

01 November 2018 / Posted in: Treatments & Research

Despite our urgent request, Biogen has not extended the cut off for this programme to 1st December. It ends now for any newly diagnosed infants. Read their community statement here.

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Update on Access to Nusinersen

26 October 2018 / Posted in: Treatments & Research

Thank you to the patient and clinical experts who strongly advocated for access to nusinersen at NICE’s committee meeting this week; to clinicians, families and adults who: expressed their views in their submissions to NICE; wrote letters to MPs, NICE, NHS England and Biogen; were there on the day.

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AveXis Community Update on Submitting their Application for AVXS-101

19 October 2018 / Posted in: Treatments & Research

AveXis yesterday submitted their regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1. This is an important initial step toward the potential approval of AVXS-101, a gene replacement therapy candidate for the treatment of the underlying root cause of SMA.

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$3 million 'Breakthrough' prize for Developers Behind Spinraza

18 October 2018 / Posted in: Treatments & Research

An article in The Guardian, published yesterday, describes how C Frank Bennett, senior vice president of research at Ionis Pharmaceuticals, and Adrian Krainer of Cold Spring Harbor Laboratory, have won the prize for developing Spinraza.

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SMA UK’s submission to NICE’s scoping consultation on AVXS-101 treatment for infants with SMA Type 1

17 October 2018 / Posted in: Treatments & Research

England’s regulatory body, NICE, has invited a wide range of clinical and patient groups to comment on this treatment. You can read what we have said to NICE here.

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