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Latest News: Research

Important safety update on SMN2 splice-modifying drug, RG7800

15 May 2015 / Posted in: Research

Roche, PTC Therapeutics, and the SMA Foundation have provided a critical update on the safety of their SMN2 splice-modifying compound, RG7800.

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SMA Europe announces funding awards

29 April 2015 / Posted in: Research

Four of the 2014 applications have been successful and SMA Support UK’s contribution will go towards one of them.

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SMA Support UK at Rare Disease Day 2015

20 March 2015 / Posted in: Research

Find out about how SMA Support UK was involved with Royal Holloway’s annual Rare Disease Day event.

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Splicing, Exons and the SMN2 'back-up' gene

19 March 2015 / Posted in: Research

Information on the importance of splicing and how it can be modified to increase SMN protein levels.

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Roche to buy Trophos

23 January 2015 / Posted in: Research

Roche, the Swiss pharmaceutical giant, has agreed to purchase the small, late-stage clinical drug testing company, Trophos, and its lead drug with potential for SMA, Olesoxime

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SMN Gene Therapy in a Large SMA Model

06 January 2015 / Posted in: Research

Prof. Arthur Burghes (Ohio State University) and colleagues have just published a study aimed at determining whether reducing SMN levels in pig motor neurons results in symptoms similar to SMA.

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Isis Pharmaceuticals Initiates Second Phase III Trial of ISIS-SMNRX

12 December 2014 / Posted in: Research

ISIS Pharmaceuticals has announced the start of a second Phase III trial of its gene therapy drug ISIS-SMNRx.

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PTC Therapeutics SMA drug enters Phase Ib/IIa trial

26 November 2014 / Posted in: Research

PTC Therapeutics has announced that its SMN2 splice-modifying drug, RG7800, has entered a Phase Ib/IIa clinical trial in adults and children with SMA.

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ISIS-SMNRx Phase II Trial Results Released

14 October 2014 / Posted in: Research

ISIS Pharmaceuticals has released detailed results from their Phase II clinical trials of the gene therapy drug ISIS-SMNRx.

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Isis Pharmaceuticals Begin Recruitment for Phase III Trial of ISIS-SMNRX

10 October 2014 / Posted in: Research

ISIS Pharmaceuticals have begun recruiting young SMA patients at a number of sites in the US for a Phase III trial of its gene therapy drug ISIS-SMNRx.

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