| 06/18/2021 | |||
| 8:00 pm - 9:00 pm | Friday Welcome - Breakout Sessions Friday Evening Syndrome-Specific Breakout Session | ||
| 9:00 pm - 10:00 pm | Friday Evening Connection-Specific Breakout Session | ||
| 06/19/2021 | |||
Session I - Welcome and Keynote | |||
| 12:00 pm - 12:20 pm | Welcome Presentation 2021 Message - Serving Families and Funding Research Lisa Todd, MPS II parent, Board of Directors, National MPS Society Terri Klein, President and CEO , ML Parent | ||
| General Main Session | |||
| 12:20 pm - 1:00 pm | KEYNOTE SPEAKER Parenting Driven by the Audacity of Hope and Love Mercedes Ramirez Johnson, Rare Disease Advocate/Transformational Leadership | ||
| 1:00 pm - 1:40 pm | MPS and ML Overview - 2021 Insights and Overview Joseph Muenzer, MD, PhD, University of North Carolina Medical Genetics | ||
| 1:40 pm - 1:45 pm | Panelist Q&A Lisa Todd, MPS II parent, Board of Directors, National MPS Society Joseph Muenzer, MD, PhD, University of North Carolina Medical Genetics Mercedes Ramirez Johnson, Rare Disease Advocate/Transformational Leadership Terri Klein, President and CEO , ML Parent | ||
| 06/19/2021 | |||
Session II - Newborn Screening, Pioneering a Second Nomination | |||
| 1:45 pm - 2:15 pm | Newborn 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 | ||
| 2:15 pm - 2:45 pm | Newborn Screening - Why Science Matters Matthew Ellinwood, DVM, PhD, National MPS Society | ||
| 2:45 pm - 3:15 pm | Working with State Advocacy to Pass Newborn Screening Legislation Dylan Simon, Everylife Foundation | ||
| 3:15 pm - 3:25 pm | Panelist Q&A 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 Matthew Ellinwood, DVM, PhD, National MPS Society | ||
| 06/20/2021 | |||
Family Conference - Session I, Sunday | |||
| 12:00 pm - 12:40 pm | Navigating Clinical Trials for MPS Paul Harmatz, M.D. | ||
| 12:40 pm - 1:10 pm | Changes in Bone and Joints after HCT and/or ERT - 10 Year MPS Observational Study Lynda Polgreen, MD, MS, Associate Professor, Lundquist Institute at the Harbor-UCLA Medical Center and Children's Hospital Orange County | ||
| 1:10 pm - 1:15 pm | Clinical Trials Q&A Paul Harmatz, M.D. Lynda Polgreen, MD, MS, Associate Professor, Lundquist Institute at the Harbor-UCLA Medical Center and Children's Hospital Orange County | ||
| 06/20/2021 | |||
Family Conference - Session II, Sunday | |||
| 1:15 pm - 1:45 pm | Natural History of MPS Disorders: What do we know (and need to know) About Cognition Over Time? Elsa G. Shapiro, PhD, ABPP, Professor of Pediatrics and Neurology, Division of Pediatric Behavioral Neuroscience, Shapiro Neuropsychology Consulting | ||
| 1:45 pm - 2:15 pm | Neurobehavioral Manifestations and Other Considerations in MPS Julie Eisengart, PhD, LP, Pediatric Neuropsychologist and Assistant Professor at the University of Minnesota | ||
| 2:15 pm - 2:25 pm | Neurocognitive Q&A; Concurrent Health Break Julie Eisengart, PhD, LP, Pediatric Neuropsychologist and Assistant Professor at the University of Minnesota Elsa G. Shapiro, PhD, ABPP, Professor of Pediatrics and Neurology, Division of Pediatric Behavioral Neuroscience, Shapiro Neuropsychology Consulting | ||
| 06/20/2021 | |||
Family Conference - Session III, Sunday | |||
| 2:30 pm - 3:00 pm | Orthopedic Management of the Mucopolysaccharidoses Klane White, MD, Seattle Childrens Hospital | ||
| 3:00 pm - 3:30 pm | Bone Marrow Transplantation and Updates for MPS Disorders Paul Orchard, M.D., University of Minnesota | ||
| 3:30 pm - 3:45 pm | Panelist Q&A Paul Orchard, M.D., University of Minnesota | ||
| 06/20/2021 | |||
Science Conference Sunday Sessions | |||
| 12:00 pm - 1:00 pm | Scientific Session I - Updates on Advances in ML Research | ||
| 12:00 pm - 12:20 pm | Cathepsin Proteases Drive Heart Valve Defects in MLII Zebrafish Heather Flanagan-Steet, PhD, Director of Functional Studies , Greenwood Genetic Center | ||
| 12:20 pm - 12:40 pm | Evaluation of systemic AAV gene therapy in the feline model of mucolipidosis II Allison Bradbury, MS, PhD, Assistant Professor, Department of Pediatrics; Principal Investigator, The Ohio State University; Center for Gene Therapy at the Abigail Wexner Research Institute | ||
| 12:40 pm - 12:55 pm | Questions and Discussion Allison Bradbury, MS, PhD, Assistant Professor, Department of Pediatrics; Principal Investigator, The Ohio State University; Center for Gene Therapy at the Abigail Wexner Research Institute Heather Flanagan-Steet, PhD, Director of Functional Studies , Greenwood Genetic Center | ||
| 06/20/2021 | |||
| 1:00 pm - 2:00 pm | Scientific Session II - Overview and Ongoing Studies of Pentosan Polysulfate as a Therapy in MPS | ||
| 1:00 pm - 1:20 pm | Pentosan polysufate for MPS Calogera (Lilla) M. Simonaro, PhD, Professor, Department of Genetics and Genomic Sciences Icahn School of Medicine at Mount Sinai | ||
| 1:20 pm - 1:40 pm | Pentosan polysulfate therapy improves neuropathological measures in the canine model of MPS IIIB Jodi D. Smith, DVM, PhD, Associate Professor, College of Veterinary Medicine at Iowa State University | ||
| 1:40 pm - 1:55 pm | Questions and Discussion Calogera (Lilla) M. Simonaro, PhD, Professor, Department of Genetics and Genomic Sciences Icahn School of Medicine at Mount Sinai Jodi D. Smith, DVM, PhD, Associate Professor, College of Veterinary Medicine at Iowa State University | ||
| 06/20/2021 | |||
| 2:00 pm - 3:00 pm | Scientific Session III - Advances in Approaches to Care and Monitoring of MPS Disease | ||
| 2:00 pm - 2:20 pm | A phase 1 clinical trial of in utero enzyme replacement therapy for fetuses with Mucopolysaccharidoses Tippi MacKenzie, MD, Professor of Surgery, University of California San Francisco | ||
| 2:20 pm - 2:40 pm | Predictors of IQ in Hurler syndrome Troy Lund, MSMS, PhD, MD, FAAP, Associate Professor, University of Minnesota Medical School | ||
| 2:40 pm - 2:55 pm | Questions and Discussion Troy Lund, MSMS, PhD, MD, FAAP, Associate Professor, University of Minnesota Medical School Tippi MacKenzie, MD, Professor of Surgery, University of California San Francisco | ||
| 06/26/2021 | |||
Clinical Trial and Industry/Sponsor Updates, Saturday Sessions | |||
| 12:00 pm - 1:00 pm | AVROBIO, Orchard Therapeutics, Sigilon | ||
| 1:00 pm - 2:00 pm | Denali Therapeutics, JCR Pharmaceuticals, REGENXBIO | ||
| 2:00 pm - 3:30 pm | Abeona Therapeutics, Biomarin, Sanofi-Genzyme, Takeda, Ultragenyx | ||
| 06/27/2021 | |||
Family Conference Main Room, Sunday Sessions (Concurrent) | |||
| 12:00 pm - 12:30 pm | Our Impact: Fundraisers Make a Difference | ||
| 12:30 pm - 1:00 pm | Genetic Counseling and Family Planning 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 | ||
| 1:00 pm - 3:00 pm | Syndrome-Specific Sessions, Live on Zoom | ||
| 1:15 pm - 2:15 pm | MPS I breakout sessions: transplant care, non-transplant care | ||
| 2:00 pm - 3:00 pm | MPS II breakout session | ||
| 1:00 pm - 1:45 pm | MPS III research updates, live presentation, and Q&A Kim Hemsley, PhD, Associate Professor, Childhood Dementia Research Group, Flinders University | ||
| 1:45 pm - 3:00 pm | MPS III breakout session | ||
| 1:15 pm - 2:15 pm | MPS IV breakout session | ||
| 1:15 pm - 1:45 pm | MPS VI/VII updates and care management presentation Paul Harmatz, M.D. | ||
| 1:45 pm - 3:00 pm | MPS VI/VII breakout session | ||
| 1:00 pm - 2:00 pm | ML updates and care management, live presentation, and Q&A | ||
| 2:00 pm - 3:00 pm | ML breakout session | ||
| 06/27/2021 | |||
Science Conference Sunday Sessions | |||
| 12:00 pm - 1:00 pm | Scientific Session I - MPS II Neuropathology in Models and Clinically Relevant Imaging | ||
| 12:00 pm - 12:20 pm | Dysregulation of axonal guidance-related molecules and defects in visual-evoked tectal responses in MPSII zebrafish model Enrico Moro, PhD, Associate Professor, University of Padova, Italy | ||
| 12:20 pm - 12:40 pm | Decoding the phenotypic variability in mucopolysaccharidosis type II Igor Nestrasil, MD, PhD | ||
| 12:40 pm - 12:55 pm | Questions and Discussion Enrico Moro, PhD, Associate Professor, University of Padova, Italy Igor Nestrasil, MD, PhD | ||
| 06/27/2021 | |||
| 1:00 pm - 2:00 pm | Scientific Session II - MPS Phenotype Prediction | ||
| 1:00 pm - 1:20 pm | Predicting Pathogenicity of IDUA and IDS Variants Richard Steet, PhD, Director of Research, Greenwood Genetic Center | ||
| 1:20 pm - 1:40 pm | Classification of IDS gene variants using saturation genome editing Patricia Dickson, MD, Professor, Washington University School of Medicine in St. Louis | ||
| 1:40 pm - 1:55 pm | Questions and Discussion Patricia Dickson, MD, Professor, Washington University School of Medicine in St. Louis Richard Steet, PhD, Director of Research, Greenwood Genetic Center | ||
| 06/27/2021 | |||
| 2:00 pm - 3:00 pm | Scientific Session III - Overviews of Neuropathology and Immune Activation in MPS Disease | ||
| 2:00 pm - 2:20 pm | Our current understanding of neuropathology in the MPS disorders Kim Hemsley, PhD, Associate Professor, Childhood Dementia Research Group, Flinders University | ||
| 2:20 pm - 2:40 pm | Anti-inflammatory Biologics: experimental adjunctive treatments of MPS Michelina Iacovino, PhD, Associate Professor, Lundquist Institute at the Harbor-UCLA Medical Center Lynda Polgreen, MD, MS, Associate Professor, Lundquist Institute at the Harbor-UCLA Medical Center and Children's Hospital Orange County | ||
| 2:40 pm - 3:00 pm | Questions and Discussion, Concluding Comments Kim Hemsley, PhD, Associate Professor, Childhood Dementia Research Group, Flinders University Michelina Iacovino, PhD, Associate Professor, Lundquist Institute at the Harbor-UCLA Medical Center Lynda Polgreen, MD, MS, Associate Professor, Lundquist Institute at the Harbor-UCLA Medical Center and Children's Hospital Orange County | ||
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