Weekly Quiz

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Rare Blood Disorder Gets New Drug Approval

The FDA has approved a new drug, 1) ___, to treat a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH). It will cost hundreds of thousands of dollars to treat a single patient. In PNH, the 2) ___ system destroys red blood cells, causing darkened urine, pain in the head, back and abdomen and an increased risk of blood clots that can lead to death by causing heart attacks and other complications. Most patients live 3) ___ years after their diagnosis. Soliris does cause an increased risk of infection in these patients, while reducing the risk of blood clots. An estimated 8,000 people in the U.S. and Europe have PNH, which is caused by a 4) ___ mutation that occurs during a patient’s lifetime and cannot be passed on to children. The strategy of treating a very rare disease with an incredibly expensive biotech medicine has been around at least since 5) ___ received approval to sell its Ceredase for Gaucher’s disease in 1991 at a widely reported cost of $150,000 a year. Genzyme has become one of the biggest and most profitable biotech companies partly because of its drugs to treat 6) ___ diseases, although it has branched out into other areas, Soliris has been tested in rheumatoid arthritis, but it is as a PNH drug that it is likely to make it to market.


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