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A total of 959 posts are filed under Insights
Hacking Rare Disease
As a teenager, Bill Paseman worked with his father in the refineries around the Houston area. He remembers remarking to him at the time that… Continue Reading
How Bo Bigelow Became a Hunter-Gatherer
Bo Bigelow is on a quest to find 68 patients. Bigelow, chairman of the Foundation for USP7-Related Diseases, needs to find 68 more patients… Continue Reading
Are Orphan Drugs Losing their Luster?
The orphan drug market is experiencing strong growth, but there are signs that pricing pressures may be impacting the sector, raising… Continue Reading
Why Rare Disease Patients Should Worry About the Shifting Discussion from Value to Scarcity
The high cost of rare disease therapies is often discussed in isolation. When a new drug is approved for an orphan indication and an… Continue Reading
Why Patient Groups Shouldn’t Dismiss Report on Pharma Influence
Earlier this month, Kaiser Health News launched “Pre$cription for Power,” a database that tracks Big Pharma contributions to patient… Continue Reading
Small Data Matters Too
We first walked through the doors of UNC Children’s Hospital in 2009, scared parents who flew several states away to see an expert on our… Continue Reading
From Horses to Zebras: My Rare Disease Journey
Author: Amanda DeBellis I’ve been sick my whole life: crippling stomach aches, chronic infections (ears, sinuses, stomach bugs, and skin… Continue Reading

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