Latest News: Research
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.
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.
20 March 2015 / Posted in: Research
Find out about how SMA Support UK was involved with Royal Holloway’s annual Rare Disease Day event.
19 March 2015 / Posted in: Research
Information on the importance of splicing and how it can be modified to increase SMN protein levels.
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
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.
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.
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.
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.
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.