I live in London
ovral g So I quickly googled “amicus” and “amenable mutation” and I think the third hit was a poster by Amicus which explains what the drug is, how it’s supposed to work, what the assay is, and NOT how many people it would be effective on (you know, all the stuff that probably should have been in your article)
http://www.amicusrx.com/pdf/WORLD 2014 FACETs Poster_EB.pdf Turns out Amicus performed their own assay using HEK cells but, when they had an independent company repeat the assay under GLP conditions, 10% of the mutations Amicus said were amenable turned out not to be (and isn’t it funny how “random” errors ALWAYS wind up being made in the company’s favor — they didn’t misidentify 10% of patients that it would work on as not being treatable, but said they could sell it to people who would not respond).