This webcast is based on a roundtable discussion on the optimal diagnosis and future management of lysosomal diseases. Three experts specializing in Fabry, Gaucher, and Pompe disease will raise key clinical questions and discuss how to overcome barriers in daily clinical practice across the lysosomal disease spectrum. The experts will provide an update on the long-term effects of ERT and highlight the remaining challenges, including the consequences of delayed diagnosis. They will discuss the role and importance of early diagnosis and treatment initiation, and provide perspectives on the recent advances in screening programs for newborn and high-risk populations. Lastly, the experts will address the position of emerging therapies and strategies for the optimal management of lysosomal diseases.
Genetics & Genomics
Elsevier's CME courses target medical geneticists with the need for the latest genetic and genomic knowledge.