This CME/CE activity, hosted by Ozlem Goker-Alpan, MD, Co-founder and President Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) and Sonata Jodele, MD, Research Professor of Pediatrics at Cincinnati Children’s Hospital Medical Center, highlights the current trends in gene therapy for lysosomal storage diseases as well as some of the safety concerns with such therapy.

At the end of this activity, participants should be able to:
Describe the limitations and unmet needs of the current therapeutic landscape for lysosomal disorders
Review the gene transfer therapies for lysosomal disorders
Describe the best clinical practices to monitor the safety profile of gene therapy
Describe the best practices for gene therapies

Educational Support for this activity was provided by Takeda, Sanofi, and Chiesi.

To obtain CME credit, please visit https://checkrare.com/learning-center/courses/