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In Rare Form

A total of 46 posts are filed under In Rare Form
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
Thinking Ahead
When the European Medicines Agency approved Myozyme in 2006, Genzyme’s enzyme replacement therapy for the lysosomal storage disorder… Continue Reading
Greater Transparency Would Improve Patients’ Understanding of FDA Actions
When the U.S. Food and Drug Administration declines to approve a new medicine, its delivers the news to the drug developer in what’s known… Continue Reading
Phrank Talk about Diagnosing Rare Diseases
I studied Latin for four years. Reading it was not a problem. Translating it, though, was another issue. Once we moved on from Tres Porci… Continue Reading
TV Show Melds Dark Humor with Unvarnished View of Life with Chronic Illness
Travis Flores grew up with cystic fibrosis, spending much time in hospitals and attending support groups in the facilities where he got… Continue Reading
Spotting Birds of a Feather
John Atkinson became a birder in fifth grader when his teacher told the students to put away their science books and follow her outside.… Continue Reading
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