How one group of researchers brought a scientific idea to the clinic for a rare disease


Scott Rollins was starting his graduate thesis at Oklahoma Medical Research Foundation in the late 1980s when he first heard about paroxysmal nocturnal hemoglobinuria (PNH). He learned that in PNH, red blood cells are vulnerable to attack by the complement system's terminal membrane-attack complex. Patients with PNH have episodes of hemolysis marked by dark urine and anemia, as well as stomach and back pain, chronic exhaustion, and an increased risk of life-threatening blood clots.

A mainstay of medical school textbooks, PNH is nonetheless rarely encountered, occurring in only 8,000 to 10,000 people in North America and Europe combined. For Rollins' graduate advisor, Peter Sims, dissecting...

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