Monday 29 July 2013

Clinical trial recruitment updates - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Clinical trial recruitment updates - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: The FOR DMD study is now open to recruitment in most UK sites: Newcastle, GOSH, Cardiff, Manchester, Leeds, Liverpool, Glasgow and Birmingham.

The FOR DMD study is an international study which evaluate the three most commonly prescribed corticosteroid regimes in DMD to establish which one is of greatest benefit and which one is associated with the best side effect profile. Participating boys will receive one of the three regimes and will be closely monitored for muscle strength, performance and development of any side effects in accordance to the highest standards of care. Further information will de desiminated and please refer to the website for more details (link below). 

Recruitment for US phase Ib and IIa studies testing halofuginone (HT-100 by HALO Therapeutics) is currently exceeding targets. This compound is the first antifibrotic to be trialled in DMD and was previously supported at the early stages by various patients organisations, including Action Duchenne. Halofuginone, interrupts signalling in a key fibrotic pathway; preventing the replacement of normal muscle fibres and stabilising muscle function. 

The HALO team are planning ahead with larger multinational pivotal phase II/III studies estimated to take place in 2015 (see link at the end for further details). 

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. 

http://www.actionduchenne.org/jsp/uploaded_files/documents/ROOT/2013-06-24%20FAQ%20FINAL%20for%20print%20ATA.pdf 


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