Pafuramidine, a US FDA–designated orphan drug, has been tested against the parasitic trypanosome disease human African trypanosomiasis ( sleeping sickness). Pafuramidine reached Phase III clinical trials for sleeping sickness; but in 2008, it was pulled because it produced liver toxicity and renal insufficiency. New preliminary clinical trials show that pafuramidine is effective against pneumocystitis pneumonia with relatively few side effects.
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