Data! In PLoS One (open access)...
Zaharieva IT, Calissano M, Scoto M, Preston M, Cirak S, Feng L, Collins J, Kole R, Guglieri M, Straub V, Bushby K, Ferlini A, Morgan JE, Muntoni F. Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy. PLoS ONE. 2013;8(11):e80263. doi:10.1371/journal.pone.0080263
From the discussion:
"Our analysis in DMD patients did not identify a significant difference when the levels of dystromirs were compared in serum samples before treatment and in post-treatment samples taken at week 12, when the last dose of eteplirsen was administered, despite an encouraging trend for normalisation of the miRNAs. Further analysis in the DMD patients according to the dose and response to eteplirsen did not reveal a conclusive result of the difference in the level of the studied miRNAs before and after treatment."
Oh well, back to the 6-minute walking test.
Interesting that at this point we only have results of these serum markers for baseline and 12 weeks. Somebody knows the results from longer duration. And somebody that would know has been buying stock in the open market.
A possible biomarker to measure success of our therapy. Done on the 12 British kids in that 2010 trial. They say that they would need to test the theory on a larger cohort that is dosed for longer than the 12 weeks of this cohort. They probably did it - are still doing it - on our present 12 kids. Could be a very neat biomarker for DMD. Chris should be asked about it - and whether we're testing it in our current group - and he may tell us if it's panning out. Seems like a very clear-cut and I'm guessing relatively easy test. Non-invasive. Hoffman should be thrilled!