MPS I Webinar: Clinical trial and treatment update

Updated: Aug 6, 2021

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.

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