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In Rare Form

A total of 58 posts are filed under In Rare Form
Fighting Emergency Medical Protocols that Endanger Rare Disease Patients
Darlene Shelton was proud of the clever bit of safety gear her family had installed in their vehicle for her grandson Danny, who has the… Continue Reading
Learning to Listen to the Patient-Consumer
Shay Brill remembers shopping at malls during store hours, standing in long lines before Christmas, and the frustration of finding a… Continue Reading
Giving New Meaning to “Translational Research”
For rare disease advocates, getting access to the latest medical research can be daunting. The high price tag makes paying for access to… Continue Reading
The What and the How
I tend to multi-task, thanks to a little help with some modest technology. During any given day I will have a long to-do list on my computer… Continue Reading
Teaching Others to See
Hours after Rachel Callander’s daughter Evie was born, the infant was having difficulty eating and began choking. The medical staff at the… Continue Reading
How Rare Disease Advocates Can Get the Most out of Collaborations
Collaborations play a critical role in advancing potential treatments for a rare disease and can take many forms. In some cases, they may… Continue Reading
Ferris Bueller and the Cost of Pivotal Clinical Trials
It turns out asking what a pivotal phase 3 clinical trial costs is a little like asking, “What does a car cost?” The answer will depend… Continue Reading

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