Proteus syndrome is a rare disorder and an orphan disease: incidence is less than one in 1 million people worldwide.
Only a few hundred individuals have been reported in medical literature.
About 50 patients are being actively followed by the NIH in a longitudinal study with the total patient population estimated to be 100 to 200 in the US.
Patient registries are available in some European countries where advocacy groups also are active.
Though patients may have minimal or no manifestations at birth, the disease develops and becomes apparent in early childhood (six-18 months) and rapidly progresses with intense growth in the first 10 years of life.
Very few patients reach adulthood.
The only available therapeutic intervention for Proteus syndrome is surgery.

Source: ArQule Leerink Swann presentation, Feb. 17, 2017

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