by Feba Sam | Dec 11, 2017 | Perlara science |
The Condition: Mucolipidosis Type IV Mucolipidosis is a group of inherited lysosomal storage disorders characterized by aberrant storage and accumulation of lipid vesicles in patient cells. Mucolipidosis type IV (MLIV) is a rare genetic lysosomal storage disorder that...
by Ethan Perlstein | Dec 6, 2017 | Perlara science |
This is the fourth post in an open science blog series about PERL101 (P101), a small molecule with big hopes. Demystifying drug discovery The default setting of biotech startups and pharma giants alike is stealth mode. At Perlara the default is set to open. As the...
by Tamy Portillo Rodriguez | Nov 1, 2017 | Perlara science |
Perlara’s high throughput screen progress in Mucolipidosis IV (MLIV) flies… In this blog post, I’ll tell you about Perlara’s high throughput screen progress in MLIV flies. Last year, Tom Hartl reviewed some details of MLIV disease biology and described how...
by Ethan Perlstein | Mar 8, 2017 | Perlara science |
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...
by Feba Sam | Feb 1, 2017 | Perlara science |
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...
by Nina DiPrimio | Jan 12, 2017 | Perlara science |
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...
