The Curious Case of PTC Therapeutics: A Surge Born of Promises and Phenylalanine

Picture, if you will, a man standing before an audience of bespectacled financiers, his voice dripping with the honeyed assurance of a merchant selling golden eggs. This man is Matthew Klein, CEO of PTC Therapeutics, who spoke not of wars or revolutions but of something far more insidious: phenylketonuria, or PKU. Imagine a condition so cruel that it forces its victims into a lifetime of dietary asceticism, banishing them from the simple joys of chocolate cake and roasted meats. And lo, here comes Sephience, their savior-an oral therapy approved both in the U.S. and Europe, capable of lowering the accursed levels of phenylalanine that build up in these poor souls.








