June in tweets

Last month’s highlight was that we completed the primary screening phase of our NPC lead program. Let’s take a closer look at how summer started at PLab.

We kicked off June with the first in a three-part blog series on what’s happening in the world of orphan drugs for monogenic diseases, starting with two disturbing trends with respect to prices and approvals.

PLabbers celebrating the beginning of fundraising season.

Nina, Tom and I attended the Ara Parseghian conference for Niemann-Pick C research in Notre Dame, IN. This will hopefully become an an annual tradition.

Here’s the poster we presented at the Parseghian meeting, which includes data on the yeast, worm and fly primary screens.

One of our most popular blog posts to date described our burn rate. As a PBC, we seek to maintain a higher standard of transparency, starting with our budget.

Our summer intern Maria has been blogging about her two projects. The first one is computational and is looking at whether disease-causing mutations preferentially occur in evolutionarily conserved amino acids. The second one is experimental and involves validating a small-molecule suppressor screen for 3 ALS models in yeast.

Sangeetha, our resident worm expert, attended the international GSA C. elegans meeting at UCLA.

 

And that’s a wrap for June!