Transforming Clinical Outcomes with Early Treatment of Lysosomal Disorders +++

https://checkrare.com/learning/p-transforming-clinical-outcomes-with-early-treatment-of-lysosomal-disorders/ Goker-Alpan, MD, David F. Kronn, MD, Uma Ramaswami, MD, Liz Jalazo, MD, and Lindsay Torrice, MD examine the evidence available to address how to monitor, and possibly treat, children with lysosomal diseases that were diagnosed by newborn screening or soon after birth. PAH Clinical Research Highlights: CHEST 2024 https://checkrare.com/learning/p-pah-clinical-research-highlights-chest-2024/ Jean Elwing, MD provides an overview of the latest clinical research about PAH. Skeletal Involvement in Lysosomal Disorders https://checkrare.com/learning/p-skeletal-involvement-in-lysosomal-disorders/ Ozlem Goker-Alpan, MD and Ravi Kamath, MD, PhD discuss best practices to identify and treat bone problems associated with lysosomal disorders.