|Rare Diseases Working Group|
Co-Chairs: Yaffa Rubinstein and Kerry Goetz
Orphan or rare diseases are a clinically heterogeneous group of over 7,000 disorders which are commonly diagnosed during childhood and often have deleterious long term effects. Their cumulative public concern around the world is substantial and depending on whose definition you apply, the incidence will range around 1-8/ 10,000. Unfortunately, only around 300 of these disorders have an effective drug therapy. In order to expand our scientific knowledge of these disorders, several biorepository issues need to be addressed.
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