PTC Therapeutics, Inc. (PTCT) Presents at Barclays 28th Annual Global Healthcare Conference Transcript

Overview of PKU and Sephience - PKU (Phenylketonuria) is a rare genetic disorder where individuals cannot metabolize the amino acid phenylalanine, leading to its accumulation in the body, which severely impacts brain development and neurological function [1] - Newborn screening for PKU is critical, allowing early diagnosis and the initiation of a highly restrictive diet to prevent protein intake, which is essential for avoiding severe health consequences [1] - There are approximately 17,000 individuals in the United States diagnosed with PKU, with an estimated 58,000 affected globally in markets targeted for Sephience's therapy [1]