34th Annual Family Conference, Virtual Event
Please note that all times listed are in Eastern Daylight Time (EDT).
8:00 pm
8:00 pm - 9:00 pmFriday Evening Syndrome-Specific Breakout Session
9:00 pm - 10:00 pmFriday Evening Connection-Specific Breakout Session
9:45 am - 10:00 amNational MPS Society - Pivoting In a New Climate
Lisa Todd, MPS II parent, Board of Directors, National MPS Society
General Main Session
10:00 am - 10:30 amKEYNOTE SPEAKER - Navigating MPS During the COVID-19 Pandemic
Heather A. Lau, MD, Assistant Professor, Department of Neurology and Director of the Lysosomal Storage Disease Program, NYU Grossman School of Medicine
10:30 am - 11:15 amGeneral MPS Overview and Clincal Trial Updates During a Pandemic
Joseph Muenzer, MD, PhD, University of North Carolina Medical Genetics
11:15 am - 11:45 amNewborn Screening: What is It? How Will It Help the Diagnostic Journey?
Barbara Burton, MD, Professor of Pediatrics (Genetics, Birth Defects, and Metabolism), Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
11:45 am - 11:50 amGeneral Main Session Q&A
11:50 am - 12:00 pmVisionary Leadership Award presented to Barbara Wedehase, MSW, GGC
Matthew Ellinwood, DVM, PhD, National MPS Society
SESSION II Management and Care for MPS and ML (Track I)
12:45 pm - 1:15 pmNatural History of MPS Disorders - What Do We Know About Cognition Over Time?
Elsa G. Shapiro, PhD, ABPP, Professor of Pediatrics and Neurology, Division of Pediatric Behavioral Neuroscience, Shapiro Neuropsychology Consulting
1:15 pm - 1:45 pmNeurobehavioral Manifestations and Otehr Considerations in MPS
Julie Eisengart, PhD, LP, Pediatric Neuropsychologist and Assistant Professor at the University of Minnesota
1:45 pm - 1:55 pmNeurocognitive Q&A; Concurrent Health Break
1:55 pm - 2:40 pmOrthopedic Management of the Mucopolysaccharidoses
Klane White, MD, Seattle Childrens Hospital
2:40 pm - 3:25 pmAirway Problems with Patients of Mucopolysaccharidoses
Robert Wood PhD, MD, Cincinnati Children's Hospital
3:25 pm - 3:30 pmOrthopedic, Pulmonary, and Advanced Care Q&A
3:30 pm - 3:55 pmMucolipidosis - Building a Research Construct
SESSION III Science in MPS and ML (Track II)
12:45 pm - 1:30 pmGene Therapy Overview
1:30 pm - 2:00 pmPharmacological Presentations
Matthew Ellinwood, DVM, PhD, National MPS Society
Session IV Industry Updates
2:00 pm - 3:55 pmIndustry Updates (Sanofi-Genzyme, Abeona Therapeutics, REGENXBIO, Denali Therapeutics, BioMarin Pharmaceuticals, JCR Pharmaceuticals, Takeda, Ultragenyx Pharmaceutical, Orchard Therapeutics)
SESSION V - Charting our Course!
4:00 pm - 4:20 pmAdvocacy - WE NEED YOU for Newborn Screening!
Christine Tippett, MPS IV Parent, Board of Directors, National MPS Society
4:20 pm - 4:40 pmFundraising - Pivoting During the Pandemic - National Bake Sale Announcement!
Larry Kirch, MPS III Parent, Board of Directors , National MPS Society
4:40 pm - 4:45 pmConclusion and Wrap-Up