Latest News: Treatments & Research

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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Roche Community Update

16 March 2018 / Posted in: Treatments & Research

Roche has provided the following community statement on RG7916 and Olesoxime investigational programs.

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Nusinersen Expanded Access Programme for SMA Type 1 Update

12 March 2018 / Posted in: Treatments & Research

NHS England has now agreed to fund all children with SMA Type 1 who have been diagnosed by age 7 months, irrespective of the number of SMN2 copies they have. This is good news and our thanks go to Professor Francesco Muntoni who has tirelessly led the advocacy for this.

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What is the Likely Timeline for any Decisions about Access to Nuisnersen in England?

28 February 2018 / Posted in: Treatments & Research

We have had a number of queries about what is happening about any Managed Access Agreement and decisions from NICE. You can read our understanding of what is most likely, here.

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