Learning Objectives:
1.) To understand the rationale for using PEGylated enzymes as replacement therapy for ADA-SCID and refractory gout, two disorders of purine metabolism.
2.) To appreciate the research path that led to their clinical development.
3.) To understand the humoral immune responses to PEG-enzymes and efforts to diminish the impact on efficacy and tolerability.
Disclosures:
Chiesi Global Rare Diseases: grant support, consultant
Orchard Therapeutics: grant support, scientific advisory board
Horizon Therapeutics: royalties on sales of Krystexxa (also to Duke University)
CME Code: QAWCAN
Location: virtual (by WebEx only)
Directions: If you are having trouble accessing WebEx, please contact Isaac Smith at isaac.smith063@duke.edu.
Streaming: Yes, via WebEx. Link: https://dukemed.webex.com/meet/is120
Meeting Number/Access Code: 470 543 135 (you will not need this if you use link above)
Call-in number: 1-650-479-3207