Wednesday, 3 July 2013

Clarity on Drisapersen breakthrough designation and Ataluren trial update - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

On the 28th of June, GSK announced that the US Food and Drug Administration (FDA) had awarded their drisapersen drug for exon 51 skipping breakthrough status. Having been in contact with the GSK team concerning it's significance, it is quite clear that the FDA will work closely with GSK to provide advice and guidance in order to review the drug in an efficient manner. Further information has also been taken from the FDA website, of which a link is included at the bottom of the page. 

PTC therapeutics have informed the Action Duchenne team that phase III trial sites will open this autumn in the UK. Their primary compound, Ataluren is a potential treatment for those with a nonsense mutation (about 13%) of the DMD population. They have produced a frequently asked question factsheet for those who would like further information - please follow the link below. 

If there are any questions regarding DMD research or treatments on the horizon, please contact Diana Ribeiro, Head of Research: 

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