The theory for the current exon skipping drugs and the approaching next generation drugs for DMD is you have to get the drugs into the boys before the inflammation starts... the longer past the start of the inflammatory process, the lesser the results.
Considering ANP’s low dosage trial, any improvement at any level will be seen very favourably by the larger bio/pharma companies spending billions on DMD treatment.. Waynesworld has previously pointed out Sarepta’s collaboration with other projects.As DMD is a progressive disease, over a 24 week period in non ambulatory boys, the deterioration is pronounced so one should rightly assume that expectations for results showing, not just improvement, but any SLOWING of disease progression will be a fantastic outcome. On the low dosage (0.4 to 1mg/kg), the 2014 ATL1102 study by ANP into toxicity in Monkeys showed that dosing up to 3mg/kg should be safe and tolerable.
See link:
https://www.google.com.au/amp/s/www.news-medical.net/amp/news/20140402/ANP-reports-positive-results-from-chronic-toxicity-study-of-ATL1102-in-multiple-sclerosis.aspx
Could it be possible that ANP packs up stumps on this trial after enrolling 5 boys and moves straight on to the next phase with a higher dose??? We can only hope so.
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ANP: Toxicity and the question on dosage levels for ATL1102
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