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09:23
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Originally posted by waynesworld:
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From the patients and parents perspective it doesn't matter whether the therapy is exon-skipping or gene therapy, whether its steroids or other anti inflammatory treatments. What actually matters is whether the medication is effective in improving quality of life and increasing life span without nasty side effects. Outside of a cure, its the ability to be self sufficient in the little things of life and gain enjoyment from daily activities that we all take for granted. So, PUL 2.0 is a proxy measurement for all those things.Performance of Upper Limb Measure: PUL 2.0 made to the PPMD Conference in June 2016. Just for some context, before PUL 2.0 there was PUL 1.2; simply put PUL 2.0 is a refinement of PUL 1.2 to improve PUL's measurement properties and ease and clarity of testing. I wanted to highlight one slide titled Studies Using PUL . Its a list of Pharma/Biotech including Sarepta who use either PUL 1.2 or PUL 2.0 in studies. Remember this is from a presentation in 2016 so it would be interesting to see an update.
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and as Dr Voit from Sarepta says 'kids should be on this as soon as they're diagnosed' to prevent muscle damage.. extending their life by double perhaps??!
