Our first MPS I webinar specifically on new treatments brought together clinical experts to answer your questions about clinical trials and treatment updates and how they may affect MPS I patients.
The agenda was:
Welcome and housekeeping rules - Sophie Thomas, Senior Head of Patient Services and Clinical Liaisons, MPS Society
Overview of therapy options for MPS I – James Davison - UK
Small Activating RNA post-transplant in MPS I - Dr Simon Jones - UK
Lentiviral Gene Therapy for MPS I – Professor Maria Ester Bernardo - Italy
Invivo Gene therapy – Dr Raymond Wang – USA
Non-viral engineered cell-based therapy – Dr Thomas Kenny - UK
IV therapy – Professor Roberto Giugliani - Brazil
Questions and summary - Sophie Thomas, Senior Head of Patient Services and Clinical Liaisons, MPS Society
Watch the full webinar here:
View the slides here:
View the handout here (please click on the image):
Find more support, information and resources to help you if you are living with MPS, Fabry or a related disease here.
If you are interested in similar webinars, please keep checking our events page.
If you have found this information helpful please consider donating to the MPS Society.
