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The latest from our blog

 

World Rare Disease Day 2017

This post is dedicated to World Rare Disease Day (WRDD) 2017. It was at the WRRD four years earlier when the value proposition for Perlara crystalized in my mind. I went to NIH that frosty last day of February in order to meet with patient advocates and ask them one...

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Optimizing my first High Throughput Screen

I, along with Nina, performed our first high-throughput screen – well really medium-throughput but high-throughput for us – on Niemann-Pick Type C (NPC) patient-derived fibroblasts a couple of months back. We wanted to do a pilot screen with the Microsource bioactives...

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NPC1 KO mouse brain study

Well it has been a little while since I wrote a post, and a lot has happened since then, which I am sure you read about in our blogs and tweets. We changed our company name, partnered with Novartis and started partnering with patient groups to engineer our organisms...

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