Session 3 | Updates in MLD Clinical Therapies


Session 3 | Updates in MLD Clinical Therapies

Register before February 5th to receive a free gift!

Friday, March 12, 2021
10:00am – 12:00pm via Zoom

Speakers & Themes Include:

  • Dr. Bobby Gaspar, Orchard Therapeutics — Gene therapy: EMA approval, FDA status, Compassionate Use
  • Dr. David Whiteman, Takeda Pharmaceuticals — Enzyme Replacement Therapy: Embolden updates
  • Dr. Albert Seymour, Homology – Clinical Trial Updates
  • Dr. Paul Orchard, University of Minnesota — Moderator

This final panel will allow industry leaders to interface with the MLD community and provide updates about their clinical therapies. Each individual will have 10 minutes to present an update about the status of their therapies (30 minutes of presentations). The remaining hour will be used for moderated discussion about what these updates mean for MLD patients and the community, with a focus on gene therapy. Topics covered will be Orchard’s gene therapy (EMA approval, FDA status, compassionate use), Takeda’s enzyme replacement therapy (Embolden updates), and Homology’s gene therapy development.


Mar 12 2021


10:00 am - 12:00 pm

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