At the end of this session, learners will be able to understand the rationale for hematopoietic stem cell transplantation in lysosomal disorders, present clinical trial data using ex vivo gene therapy in combination with hematopoietic stem cell transplantation, and key drug therapies.
This seminar focuses on neurology and was delivered at the Open Medical Institute in Salzburg, Austria, from March 3 – 9, 2019.
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