Saturday - 09/30/2023

Family Conference  |

Care Management for MPS and ML | Breakfast and Lunch Symposiums

7:15 am – 7:45 am

Regency Ballroom

Breakfast Symposium  - Annual General Membership Meeting,

Lisa Todd Board Chair and Board of Directors

7:45 am – 8:00 am

Legacy Award, Clinician – Joseph Muenzer, PhD, MD

Chairman Award – Larry Kirch

Directors’ Award – Stephanie Cozine

8:00 am – 11:30 am

Regency Ballroom

Session I – Care Management for MPS and ML

8:00 am – 8:45 am

An ML II/III and MPS Clinical Overview and Clinical Trial Updates,

Joseph Muenzer, MD, PhD Bryson Distinguished Professor in
Pediatric Genetics and a Professor in the Department of Pediatrics
and Department of Genetics at the University of
North Carolina at Chapel Hill

8:45 am - 9:30 am

Expanding the Understanding of Function in MPS: Neurocognitive

Endpoints and Beyond, Julie Eisengart, PhD University of Minnesota

9:30 am - 10:15 am

ENT and Airway Issues Associated with MPS, David Molter, MD,
Washington University School of Medicine in St. Louis

10:15 am – 10:45 am

Cardiac Considerations and Updates, Elizabeth Braunlin, MD

University of Minnesota

10:50 am - 12:00 pm

Regency Ballroom

Session II – Newborn Screening and Understanding Different
Therapeutic Approaches

10:50 am - 11:25 am

Newborn Screening – Updates on Diagnostics in Newborns,
Barbara Burton, MD Lurie Children’s Hospital

11:25 am – 12:00 pm

Understanding Gene and Cell Therapies, Paul Harmatz, MD UCSF

12:00 pm - 1:00 pm

Regency Ballroom

Lunch Symposium – Part II Industry Updates

12:00 pm - 12:15 pm

JCR Therapeutics, TBA

12:15 pm - 12:30 pm

Orchard Therapeutics, Hematopoietic Stem Cell Gene Therapy
Studies in MPS I-H and MPS IIIA Updates
Annamarie Dillion, Orchard Therapeutics

12:30 pm - 12:45 pm

Ultragenyx, Deborah Marsden, MD Global Medical Expert Genetic
Metabolic Disorder

12:45 pm – 1:00 pm

BioMarin Pharmaceuticals, BioMarin’s Partnership with the
MPS Community, Kate Delaney, Senior Director Global Patient Affairs

1:00 pm - 2:15 pm

Regency Ballroom

Session III – Unmet Needs in Research

1:00 pm - 1:35 pm

Mucolipidosis Understanding the Research, Patricia Dickson, MD
Washington University School of Medicine in St. Loui

1:35 pm - 2:15 pm

Sanfilippo – Advancing Clinical Studies to Therapies, What Can
We Do?  Matthew Ellinwood, DVM, PhD National MPS Society

2:15 pm - 2:25 pm

Question and Response

2:30 pm - 3:00 pm

Regency Ballroom

Special Session – Family Planning and Genetic Counseling
Barbara Burton, MD, Lurie Children’s Hospital

3:00 pm - 3:15 pm

Regency Foyer

Health Break

3:15 pm - 5:15 pm

Regency Ballroom

Session IV – Warriors Navigating Life; Change Agents of
Ecosystems

Friendraising Award – Marielle Marinoff

3:20 pm - 3:40 pm

Taking Control: Fundraising for Critical Research, Marielle Marinoff
 MPS II Mother

3:40 pm - 4:05 pm

Strengthening Self-Reported Health Outcomes and Quality of Life
Through Movement, Jennifer Klein, MS, ML III Patient

4:05 pm - 4:25 pm

How to Share Your Story: Making an Impact on Capitol Hill
Mercedes Johnson, Board of Directors

4:25 pm - 5:10 pm

State Advocacy Workshop, Stephanie Cozine, Board of Directors
and Carl Kapes, Board of Directors

5:30 pm - 6:00 pm

Grass / Outdoors

Remembrance Ceremony

7:00 pm - 9:00 pm

Regency Ballroom

National MPS Society – Banquet