6.2.2017
Clinical trials for rare disease therapies face many challenges. Some of these include the small patient population on which they draw,… Continue Reading
5.30.2017
By Stacey Philpot “Not to be mean, Mommy, but Daddy is nicer than you.” These are the words my five-year-old daughter graces me with as… Continue Reading
5.26.2017
For many rare disease patients, the drug discovery and development process moves slower than the diseases they are battling. Earlier this… Continue Reading
5.19.2017
Last month the U.S. Food and Drug Administration granted Enzyvant both Breakthrough Therapy Designation and the newly established… Continue Reading
5.12.2017
There is increasing recognition of the important role patients can play by providing their insights into the drug discovery and… Continue Reading
5.6.2017
By Stacey Philpot Like a bird I soared, high above the clouds, soaking in the sun, inhaling the sheer goodness of life. I breathed in… Continue Reading
5.5.2017
Porphyria is a rare and intensely painful blood disease. Because it can manifest itself with symptoms similar to those caused by far more… Continue Reading
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