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). 

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