The problem for Prosensa is that their drug caused over 80% of the patients to have an AE. The dosage for Eteplirsen patients were 5-10x's that amount with no AE. It doesn't appear that Prosensa can increase the dosage without causing even more harm to the patients. Perhaps the dosage was too small for Prosensa to see a benefit but they can't increase it to see if that's true given the AE's.
Based on limited dystrophin data from Drisapersen, there is probable cause to think certain patients didn't get exon skipping. Additionally, two separate ph2b studies have showed trend favoring higher dose group: continuous 6mg/kg dosing regimen vs intermittent dosing regimen reported earlier this year; 6mg/kg vs 3mg/kg dosing regimen US study mentioned in cc today but not in PR. We'll see full results from all studies soon except more detailed dystrophin data.
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