Sung-Yun Pai

Sung-Yun Pai, a clinician-researcher specializing in Wiskott-Aldrich syndrome, shares vital insights on the latest treatment advancements. He discusses the evolving risk-benefit calculus in therapies due to safer options and longer follow-ups. The conversation highlights challenges like late diagnosis, donor matching, and limited access to gene therapy. Pai stresses the urgency of early referrals to specialized centers and deliberates on the decision-making process between transplant and gene therapy, emphasizing the complexities of achieving full correction.