Advances in Gene Therapy for Lysosomal Diseases

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/Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.