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

Session date: 
03/30/2021 - 8:00am to 9:00am EDT

Please login or register to take this course.
Room Number: 
WebEx only
Speaker Name: 
Dr. Michael Hershfield