FM .. a great introduction by Dame K to the science. I was busy when this happened on Wednesday and would recommend as FM suggests listening to Prof Kay's talk which explains what we are actually all here for. GLA.
Great to see this also finds has some media coverage. I note that the end of the article specifically mentions the probability that Dame K's work will also help Becker muscular dystrophy ... which I've not realised effects the same number of patients as DMD.
Thanks Gerry that was great to listen to. Great explanation of the trial and end points they hope to achieve. Key points I take is that they are confident Utrophin upregulation will work and they shown the distribution of Utrophin in animal models to be uniform through the fibre. Just need to show that in humans now. All very exciting.
And yes I completely agree that a deal (or deals) must be on the table for Ridinilazole and considering the outstanding results and benefits that deal should… Read More
"A new treatment for Duchenne muscular dystrophy could be available within five years.
Professor Kay Davies, an Oxford University researcher, has said trials of a drug to increase levels of the protein utrophin, to maintain the muscles, would start later this year and could help sufferers worldwide."
Follow the link from the Action Duchenne website for the full article Bonne… Read More
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