Category: Science

Biologics and you

Biologics and you

In our last post we talked specifically about orphan drugs that are small molecules. In this post I’ll address the orphan drugs that are biologics. They behave differently: There’s a possible increase in cost/year around 2006 but it doesn’t seem to remain...
Orphan drug prices and approvals

Orphan drug prices and approvals

This post kicks off a series about drug prices in the orphan drug space. We initially wanted to get a sense of orphan drug price trends over the last 30 years — since the passage of the Orphan Drug Act of 1983. There were a few published industry reports...
Humanized yeast to the rescue

Humanized yeast to the rescue

There are many research examples of yeast genes that were replaced by human orthologs for protein characterization, some of which with direct human disease relevance. One excellent example is out of Berkeley by researchers from the Rine lab published in 2012 where...
World Orphan Drug Congress recap

World Orphan Drug Congress recap

I attended my first World Orphan Drug Congress last week in Washington, D.C. It was a lovely time to be in DC, cherry blossoms in the air. The meeting itself was held at the Hilton in the heart of downtown. With over 700 attendees (or so I was told), there was...
Data data everywhere

Data data everywhere

I recently went to a conference where there was a panel discussion titled ‘Data Driven Research/Integrative Biology’. The talk was what you would expect it to be: a group of bioinformaticians fielding very vague questions regarding cultural truisms of the Data...
NPC in a dish

NPC in a dish

For those who have read our posts before and follow us on Twitter, you know we are using various model organisms such as fly, yeast and worm to discover novel therapeutics for a subset of rare genetic diseases. But once we find those potential drug candidates, how do...
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