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Latest News: Treatments & Research

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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SMA Support UK at the International Scientific Congress on SMA 2018

27 February 2018 / Posted in: Treatments & Research

Organised by SMA Europe with Brunhilde Wirth as the Scientific Chair, the first International Scientific Congress on SMA was held at Jagiellonian University in Kraków, Poland from 25th-27th January 2018. More than 100 laboratory and clinical SMA studies were presented by researchers from across the world to over 450 attendees.

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Biogen Releases Statement on CHERISH Data and Plans for New Clinical Research

21 February 2018 / Posted in: Treatments & Research

Biogen has provided a community statement on the final results from CHERISH, a Phase 3 study of SPINRAZA.

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