"The market is small? We know that. Yet we did not miss the fact that January 30, 2013, the day Kynamro's was granted approval, will be recorded in the textbooks of pharmaceutical sciences as the day the first systemically administered antisense therapeutic has been approved by the FDA"
This is potentailly a big deal not because of this particular drug, which has serious side affects and is treating something very rare.
But because it is the first approved drug based on RNAi, which can potentially be used to treat all sorts of things, from genetic disorder, alzheimers, and HIV infection.
Potentially, anything where we can say this gene is involved and lowering the amount of its product would help, you could treat.