Rare Diseases

At present, there are over 6,000 rare diseases reported in the United States. A rare disease is a condition with a prevalence of fewer than 200,000 persons in the United States.

While many rare diseases are little known and affect only a small number of people, the overall impact is large, with an estimated 25 million affected. Since few people are affected, there is marginal interest in the investment of capital in research for cures and therapies. Thus, the progress of such research proceeds at a snail's pace, while the affected children have little hope for breakthroughs in their lifetimes.