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Latest News: Treatments & Research

Roche Community Update: Olesoxime

30 May 2018 / Posted in: Treatments & Research

Due to negative long-term effects, Roche have decided to halt further development of their drug olesoxime. They have provided this community statement.

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Registration for the Public Gallery for the 27th June NICE Committee meeting

25 May 2018 / Posted in: Treatments & Research

This opens on 30th May and closes 13th June. The meeting will discuss access to nusinersen and be held in Manchester. We understand that there are only 30 places available.

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Biogen's Response to Charities' Letter

23 May 2018 / Posted in: Treatments & Research

On 16th May, the SMA charities wrote to leading managers of Biogen urging them to review their pricing and resubmit their application with the aim of Spinraza access being extended to those with SMA Types 2 and 3. Read Biogen's response.

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New International Standards of Care for SMA

22 May 2018 / Posted in: Treatments & Research

You can now read the new recommendations as to what assessments and interventions families should expect to find in any neuromuscular centre. A family-friendly version is being worked on now.

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Charities Send Joint Letter to Biogen

16 May 2018 / Posted in: Treatments & Research

Today, we have written to leading managers of Biogen about the SMC's decision that due to pricing, nusinersen may only be accessed by those with SMA Type 1. We are urging Biogen to review their pricing and resubmit their application with the aim of access being extended to those with SMA Types 2 and 3.

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What’s Happening with UK SMA Patient Databases?

14 May 2018 / Posted in: Treatments & Research

Biogen, has recently announced an investment of £450,000 over 3 years to further facilitate and accelerate the ability to bring together the current databases and to expand the programme to include more UK centres.

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AveXis Issues Community Statement on the SPR1NT Trial

08 May 2018 / Posted in: Treatments & Research

AveXis, the gene therapy company developing a new approach to treat SMA known as AVXS‐101, recently treated the first patient in a new study known as SPR1NT. They have provided the following community statement.

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Biogen Community Update re SMC Routine Funding Recommendation

07 May 2018 / Posted in: Treatments & Research

Following the Scottish Medicines Consortium's announcement of recommending nusinersen to be funded only for children with SMA Type 1, Biogen has released the following community update.

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Scottish Medicines Consortium (SMC) Decision and Next Steps on Access to Nusinersen

07 May 2018 / Posted in: Treatments & Research

The SMC has just recommended nusinersen to be funded only for children with SMA Type 1. Whilst we recognise this is a positive step forward, it is hugely disappointing that children, young people and adults with SMA Type 2 or Type 3 who wish to access, and could potentially benefit from, this treatment still don’t have this opportunity. We will now be pushing for a reassessment of the treatment. You can help.

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Novartis Agrees $8.7 Billion Deal to Acquire AveXis

03 May 2018 / Posted in: Treatments & Research

One of the largest pharmaceutical companies in the world, Novartis, has entered an agreement to purchase the relatively small gene therapy company, AveXis, which has been instrumental to the development and testing of the SMA gene therapy, AVXS-101.

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