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Latest News: Treatments & Research

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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Scholar Rock Experimental Drug for SMA Granted Orphan Drug Status

03 May 2018 / Posted in: Treatments & Research

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SRK-015 for the treatment of SMA. SRK-015 is the lead drug candidate of pharmaceutical company Scholar Rock.

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SMC Nusinersen Decision Timeline

09 April 2018 / Posted in: Treatments & Research

The Scottish Medicines Consortium (SMC) decision on whether it will recommend that the Scottish NHS funds nusinersen and, if so, for whom, will be published on the SMC website after 2pm on Monday 7th May.

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Nusinersen Treatment in Later Onset SMA - Results of the CHERISH Trial

28 March 2018 / Posted in: Treatments & Research

In February 2018, the prestigious New England Journal of Medicine published the results of the Nusinersen trial known as the ‘CHERISH trial’. This was a study that looked at the effects of giving Nusinersen to 126 children with later onset SMA.

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Patient Presentation at the SMC PACE Meeting

19 March 2018 / Posted in: Treatments & Research

Our thanks to two parents who shared their personal stories at the Scottish Medicines Consortium (SMC) Patient and Clinical Engagement (PACE) meeting. Their testimonies were very powerful and moving and we are very grateful to them for taking this on.

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Survey and Submission to NICE about Access to Nusinersen

19 March 2018 / Posted in: Treatments & Research

Thank you to all of you who responded to our survey about the impact of SMA and views about access to nusinersen. You can read both the results of this and our submission to NICE here.

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