How I Treat Wiskott-Alrich syndrome

How I Treat Wiskott-Alrich syndrome

Author: American Society of Hematology October 2, 2025 Duration: 13:16

In this How I Treat podcast episode, Laura Michaelis, MD interviews Sung-Yun Pai, MD about their recently published article in Blood journal "How I treat Wiskott-Alrich syndrome". They highlight recent updates in treatment, including new risk-benefit calculations due to safer treatments and longer follow-ups. Challenges include late diagnosis, lack of well-matched donors, and limited gene therapy availability. They emphasize the importance of early referral to specialized centers and the need for discussions about curative intent therapies, including transplant and gene therapy. The conversation also covers the complexities of gene therapy, such as the need for better conditioning agents and the challenges of achieving full correction in all cell types.


Produced by the American Society of Hematology, the Blood Podcast serves as an audio companion to the groundbreaking research found in its namesake journal. Each episode delves into the latest published studies, translating complex findings on blood disorders, cancers, and immune system functions into accessible discussions. You'll hear directly from the researchers and clinicians who are shaping the field, as they break down their methodologies, interpret results, and explore the real-world implications for patient care. This isn't just a summary; it's a critical conversation that examines the how and why behind the science, offering context that the printed page alone cannot provide. Whether you're a practicing hematologist, a trainee in the field, or simply fascinated by the intricate world of human biology, this podcast provides a vital stream of knowledge. It connects the dots between laboratory discovery and clinical practice, all while exploring topics that span from foundational biology to cutting-edge therapeutic advances. Tune in for a thoughtful analysis that makes the latest hematology research comprehensible and immediately relevant to your work or curiosity.
Author: Language: English Episodes: 100

Blood Podcast
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