Positive Interim Results from Phase I Trial Announced by Scholar Rock
Scholar Rock have announced positive interim results from an ongoing Phase I trial in healthy volunteers of their lead compound, SRK-015.
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Rare Diseases: Open Letter to the UK Government
Over 125 patient organisations, including SMA UK, have signed an open letter calling on the UK Government to review and refresh, as a matter of urgency, the UK Strategy for Rare Diseases before the end of 2020.
Read full storyThe Financial and Work-Related Impact of Living with SMA Type 1, 2 or 3 for Unpaid Carers
Thank you to all who responded to this survey, and to Wickenstones for their analysis. The results and the results of our survey last year about the impact of SMA are now available and are being used to inform Biogen’s continuing work with NICE re: access to nusinersen. Read the results summary here.
Read full storySubject to successful sign off, nusinersen is expected to be routinely available in Scotland for those with SMA Types 1, 2 and 3 from April 2019
Nusinersen has been routinely available to children with SMA Type 1 in Scotland since May 2018. This will continue. The SMC has now granted nusinersen ultra-orphan designation, allowing the treatment to be reviewed for those with SMA Types 2 and 3 through its new ultra-orphan appraisal process with the prospect of it being made available for at least three years while further outcome data is gathered.
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