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.
