UK to fast track. Two links, one from UK National paper and one is a (brief and not sure can view outside UK) TV interview with DMD patient and CEO of Summit
http://www.bbc.co.uk/tv/bbcone/live (at 08.50)
Who could benefit from the scheme?
Disease area: Duchenne muscular dystrophy, a rare genetic muscle-wasting disease which affects young boys. Patients lack a gene which controls muscle regeneration, so they gradually lose their physical abilities and typically die in their mid-20s. There is currently no cure.
Who’s developing it? Summit, a biotech company based near Oxford.
What does it do? It activates an alternative gene for muscle regeneration which is normally “switched off”. The medicine is based on research by the renowned geneticist Dame Kay Davies at the University of Oxford.
Why is it promising? Summit has already shown that the concept works in animal studies and has completed tests for safety in a small group of patients at Great Ormond Street Hospital. It is currently running tests measuring the levels of the drug in patients’ blood over time, and hopes to move onto a broader study to test its effectiveness later this year.
Thank you Steven, i hope that will be so....
I can not watch the Video:-(
David, I think you will find that it's just the phase 1 that's in the UK. The 52 boy phase 2 will be in the UK and the US. However, I'm not sure it matters where it's done (unless you are on the trial) as any cure, like the disease, does not recognize borders! I would also say that if you only fund things that go on in your backyard, you are rather limiting your options and If you take that further, maybe you should only fund things if it happens in your state, or maybe only in your town, or maybe in your street!
No,in UK is just phase 1,but phase 2 will also be in USA