Blue Matter Blog

The Rarest of Them All: Why Neglected Rare Diseases Require a Very Different Mindset
April 1, 2020

Introduction In previous articles, we explored several important aspects of rare diseases (RDs).  First, we showed how different authorities define the term “rare disease” (or “orphan disease”).  We also established a spectrum of rarity, ranging from those diseases that are considered common—with well-established treatment paradigms—to those that are “hyper-orphan,” affecting perhaps a couple of people …