| No location | |||
| 08/04/2022 | |||
| 7:30 am - 12:00 pm | Two Rivers | Board of Directors Meeting | |
| 2:00 pm - 8:00 pm | Hermitage Lobby | Registration | |
| 2:45 pm - 9:45 pm | Hermitage A/B | Camp Courage Opens | |
| 3:00 pm - 3:05 pm | Hermitage E/F | Welcome Announcements [More Info] Moderator: Lisa Todd, MPS II Parent, Chairman of the Board, National MPS Society | |
| 3:00 pm - 3:30 pm | Hermitage E/F | Opening Keynote Speaker - Mark Sands, PhD [More Info] Mark Sands, PhD, Professor in the Departments of Medicine and Genetics, Washington University School of Medicine | |
| 3:00 pm - 10:00 pm | Hermitage Lobby | Reflection Area | |
| 3:30 pm - 4:00 pm | Hermitage E/F | Science Leading to New Therapies for MPS [More Info] Paul Harmatz, MD, Pediatric Gastroenterologist, UCSF Benioff Children's Hospitals | |
| 4:20 pm - 4:50 pm | Hermitage E/F | Newborn Screening Updates [More Info] Barbara Burton, MD, Professor of Pediatrics - Genetics, Birth Defects, and Metabolism, Northwestern University | |
| 4:50 pm - 5:15 pm | Hermitage E/F | MPS I and MPS II Newborn Screening State by State Advocacy [More Info] N. Matthew Ellinwood, DVM, PhD, Chief Scientific Officer, National MPS Society | |
| 5:00 pm - 8:00 pm | Hermitage Lobby | MPS Music City Run, Rock and Roll 5K Package Pick-Up | |
| 5:15 pm - 6:00 pm | Hermitage E/F | Foundations Roundtable - Collaborations to Help the Patient Community [More Info] Terri Klein, MPA, President and CEO, National MPS Society Melissa Hogan, JD, MPS II Parent, Founder of Project Alive Cara O’Neill, MD, Co-Founder and Chief Science Officer, Cure Sanfilippo Foundation | |
| 6:00 pm - 7:30 pm | Hermitage C/D | Welcome Buffet Dinner | |
| 6:00 pm - 7:30 pm | Hermitage E/F | Newly Diagnosed Family Dinner (diagnosed since December 2019) Overview and Coping with MPS and ML | |
| 7:30 pm - 8:30 pm | Belle Meade | MPS I - By Syndrome Breakout Session | |
| 7:30 pm - 8:30 pm | Hermitage E/F | MPS II - By Syndrome Breakout Session | |
| 7:30 pm - 8:30 pm | Oaklands | MPS III - By Syndrome Breakout Session | |
| 7:30 pm - 8:30 pm | Belmont | MPS IV - By Syndrome Breakout Session | |
| 7:30 pm - 8:30 pm | Edgewood | MPS VI - By Syndrome Breakout Session | |
| 7:30 pm - 8:30 pm | Cheekwood | MPS VII - By Syndrome Breakout Session | |
| 7:30 pm - 8:30 pm | Kingsley | ML II/III - By Syndrome Breakout Session - Mercedes Ramirez Johnson, Moderator | |
| 7:30 pm - 9:30 pm | Evergreen | Sibling Breakout Sessions | |
| 8:30 pm - 9:30 pm | Hermitage E/F | Family Breakout: Mothers | |
| 8:30 pm - 9:30 pm | Belle Meade | Family Breakout: Fathers | |
| 8:30 pm - 9:30 pm | Belmont | Family Breakout: Grandparents | |
| 8:30 pm - 9:30 pm | Edgewood | Family Breakout: Adults with MPS/ML | |
| 8:30 pm - 9:30 pm | Cheekwood | Family Breakouts: Bereaved Families | |
| 9:45 pm | Hermitage A/B | Camp Courage Closes | |
| 08/05/2022 | |||
| 6:30 am - 8:00 am | Hermitage C/D | Breakfast Buffet | |
| 7:45 am | Hermitage A/B | Camp Courage Opens | |
| 7:45 am - 12:00 pm | Hermitage E/F | Session I - MPS and ML Management and Care | |
| 7:45 am - 8:00 am | Hermitage E/F | 2022 Fundraiser Award - Kimber Heiling, MPS VI Mother [More Info] Moderator: Lisa Todd, MPS II Parent, Chairman of the Board, National MPS Society Moderator: Steve Holland, MPS I Parent, Vice Chairman of the Board, National MPS Society | |
| 7:45 am - 8:00 am | Hermitage E/F | 2022 Chairman Award - Rev. Wayne Eppehimer [More Info] Moderator: Lisa Todd, MPS II Parent, Chairman of the Board, National MPS Society Moderator: Steve Holland, MPS I Parent, Vice Chairman of the Board, National MPS Society | |
| 8:00 am - 8:45 am | Hermitage E/F | Overview and Treatment Status of MPS and ML II/III [More Info] Joseph Muenzer, MD, PhD, Bryson Distinguished Professor in Pediatric Genetics; Professor, Departments of Pediatrics and Genetics, University of North Carolina at Chapel Hill | |
| 8:45 am - 9:30 am | Hermitage E/F | Orthopedic Updates in MPS [More Info] Klane White, MD, Director, Skeletal Health Dysplasia; Medical Director, Orthopedic Research; Professor, Orthopedic Surgery, Seattle Childrens Hospital | |
| 9:30 am - 10:00 am | Hermitage E/F | Anesthesia and Airway Complications [More Info] Paul Harmatz, MD, Pediatric Gastroenterologist, UCSF Benioff Children's Hospitals | |
| 10:00 am - 10:30 am | Hermitage E/F | Blood Stem Cell Transplantation & Gene Therapy for Hurler Syndrome [More Info] Paul Orchard, MD, Medical Director, Inherited Metabolic and Storage Disease Program; Professor, Department of Pediatrics - Blood and Marrow Transplant & Cellular Therapy, University of Minnesota | |
| 10:40 am - 11:00 am | Hermitage E/F | Q & A Session | |
| 11:00 am - 11:45 am | Hermitage E/F | Functional outcomes in MPS: Neurocognitive Function and Beyond [More Info] Julie Eisengart, PhD, LP, Associate Professor, Department of Pediatrics; Director, Neurodevelopmental Program in Rare Disease, University of Minnesota | |
| 11:45 am - 1:00 pm | Hermitage C/D | Lunch Buffet | |
| 12:15 pm | Hermitage A/B | Camp Courage Closes | |
| 12:45 pm | Hermitage A/B | Camp Courage Opens | |
| 12:45 pm - 2:45 pm | Hermitage E/F | Session II - Patient and Family Warriors | |
| 12:45 pm - 1:05 pm | Hermitage E/F | 2022 Directors' Award - Christine Tippett, MPS IV Mother, Board of Directors [More Info] Moderator: Lisa Todd, MPS II Parent, Chairman of the Board, National MPS Society Moderator: Steve Holland, MPS I Parent, Vice Chairman of the Board, National MPS Society | |
| 12:45 pm - 1:05 pm | Hermitage E/F | Visionary Leadership Award - Dr. Elizabeth F. Neufeld, PhD [More Info] Moderator: Lisa Todd, MPS II Parent, Chairman of the Board, National MPS Society Moderator: Steve Holland, MPS I Parent, Vice Chairman of the Board, National MPS Society | |
| 1:00 pm - 1:45 pm | Hermitage E/F | Adults with MPS or ML Panel [More Info] Laynie Holland, MPS I Jason Madison, MPS II Sheri Wise, MPS IVA, Board of Directors, National MPS Society | |
| 1:45 pm - 2:15 pm | Hermitage E/F | Mission Fundraising - Get Involved [More Info] Tracy Kirby, Development Director, National MPS Society Alicia Bohley Tamara Cullere Patricia Espinal, MPS VI Kimber Heiling | |
| 2:15 pm - 2:45 pm | Hermitage E/F | Siblings of MPS and ML Patients [More Info] Rebekah Holland Jasmine Henriquez Gabriela Diaz | |
| 2:45 pm - 3:00 pm | Hermitage Lobby | Health Break | |
| No location | Session III - Industry and Clinical Trial Updates | ||
| 3:00 pm - 3:15 pm | Hermitage E/F | MPS Family Conference:Takeda Update [More Info] Anthony Frangi, Vice President and Head of Takeda's US Lysosomal Storage Disorder & Endocrinology Franchise | |
| 3:15 pm - 3:30 pm | Hermitage E/F | JCR Presents Latest Updates on Pabinafusp alfa, JR-171, JR-441, and JR-446 [More Info] Mathias Schmidt, PD PhD, VP, Clinical Development, Global Business Strategy, Business Development, JCR Pharmaceuticals | |
| 3:30 pm - 3:45 pm | Hermitage E/F | REGENXBIO Interim Analysis of Data From the RGX-121 Gene Therapy Clinical Trial for the Treatment of Severe MPS II [More Info] Dawn Phillips, Director of Clinical Outcomes Research , REGENXBIO | |
| 3:45 pm - 4:00 pm | Hermitage E/F | Sanofi presents: All About MPSI Disease; Information for Patients, Families and Friends [More Info] Christopher Ortiz Courtney Zawanda | |
| 4:00 pm - 4:15 pm | Hermitage E/F | Homology New One-Time Approach to Treating MPS II: HMI-203 Investigational Gene Therapy [More Info] CeeCee Fairley M.S., L.C.G.C., Medical Affairs at Homology Medicines, Homology Medicines | |
| 4:15 pm - 4:30 pm | Hermitage E/F | Ultragenyx MPS Updates [More Info] Deborah Marsden MBBS, FACMG, Global Medical Expert , Ultragenyx | |
| 4:30 pm - 4:45 pm | Hermitage E/F | Denali Therapeutics Update on DNL 310 Clinical Program [More Info] Jeffrey M. Harris, MD, PhD, Denali Therapeutics | |
| 4:45 pm - 5:00 pm | Hermitage E/F | Orchard Therapeutics Presents Hematopoietic Stem Cell Gene Therapy [More Info] Angela Tom, MS FNP, Director, US Medical Affairs, Orchard Therapeutics | |
| 5:00 pm - 5:15 pm | Hermitage E/F | BioMarin's Partnership with the MPS Community [More Info] Guillermo Seratti, MD, BioMarin | |
| 5:45 pm | Hermitage A/B | Camp Courage Closes | |
| 6:30 pm | Downtown Nashville | Nashville Night on Your Own | |
| 08/06/2022 | |||
| 6:30 am - 11:30 am | Centennial Park, Nashville TN | National MPS Society Walk and Roll 5K Run and 1 Mile Walk | |
| 6:30 am - 7:45 am | Hermitage Lobby | Grab and Go Breakfast and Board Buses | |
| 8:00 am - 11:00 am | Centennial Park, Nashville TN | 5K Chip Timed Run | |
| 8:00 am - 11:00 am | Centennial Park, Nashville TN | Family Walk | |
| 8:00 am - 11:00 am | Centennial Park, Nashville TN | Celebration Lunch | |
| 10:45 am - 11:30 am | Centennial Park, Nashville TN | Board Buses Back to Sheraton; Return to Conference Sessions | |
| Hermitage E/F | Scientific Conference, Saturday Noon - 4:30 pm | ||
| 08/06/2022 | |||
| 12:00 pm - 12:10 pm | Hermitage E/F | Welcome, Overview, and New Developments in Science of MPS and at the Society [More Info] N. Matthew Ellinwood, DVM, PhD, Chief Scientific Officer, National MPS Society | |
| 12:10 pm - 1:00 pm | Hermitage E/F | Session I: Society Funded Research Updates [More Info] N. Matthew Ellinwood, DVM, PhD, Chief Scientific Officer, National MPS Society Jodi D. Smith, DVM, PhD, Associate Professor, Department of Veterinary Pathology, College of Veterinary Medicine at Iowa State University | |
| 12:10 pm - 12:30 pm | Hermitage E/F | A Clinically Relevant MPS IVA Animal Model [More Info] Nicola Brunetti-Pierri, MD, Associate Investigator and Head of Translation Incubator, Professor, Department of Translational Medicine, TIGEM Italy, University of Naples "Federico II' | |
| 12:30 pm - 12:50 pm | Hermitage E/F | Early Development of MPS IIIA Dopaminergic Neurons: At the Nexus of Behavior Changes and Therapy [More Info] Elvira De Leonibus, PhD, Head of Behavioral Core; Principal Investigator, Neuropsychopharmacology Laboratory, TIGEM, Italy; Institute of Biochemistry and Cellular Biology, National Research Council | |
| 12:50 pm - 1:00 pm | Hermitage E/F | Question and Answer Panel | |
| 1:00 pm - 1:50 pm | Hermitage E/F | Session II: Society Funded Research Updates [More Info] N. Matthew Ellinwood, DVM, PhD, Chief Scientific Officer, National MPS Society Jodi D. Smith, DVM, PhD, Associate Professor, Department of Veterinary Pathology, College of Veterinary Medicine at Iowa State University | |
| 1:00 pm - 1:20 pm | Hermitage E/F | Retracing the Developmental Roots of MPS II Neuropathology [More Info] Enrico Moro, PhD, Associate Professor, Department of Molecular Medicine, School of Medicine and Surgery, University of Padova, Italy | |
| 1:20 pm - 1:40 pm | Hermitage E/F | Autoimmune Component of Sanfilippo Syndrome [More Info] Michelina Iacovino, PhD, Investigator, The Lundquist Institute; Assistant Professor of Pediatrics, Division of Medical Genetics, David Geffen School of Medicine at UCLA | |
| 1:40 pm - 1:50 pm | Hermitage E/F | Question and Answer Panel | |
| 2:10 pm - 3:20 pm | Hermitage E/F | Session III: Fellow Research in the MPSs [More Info] N. Matthew Ellinwood, DVM, PhD, Chief Scientific Officer, National MPS Society Mark Sands, PhD, Professor in the Departments of Medicine and Genetics, Washington University School of Medicine | |
| 2:10 pm - 2:30 pm | Hermitage E/F | Preliminary Examination of Oligodendrocytes in the MPS VII Mouse Model [More Info] Geoffrey Eill, PhD, Postdoctoral Fellow, Laboratory of John H. Wolfe, DVM, PhD, Children’s Hospital of Philadelphia | |
| 2:30 pm - 2:50 pm | Hermitage E/F | Cognitive Function in MPS Phenotypes That Do Not Have Decline [More Info] Brianna Yund, MS, PhD, Assistant Professor of Pediatric Neuropsychology, Department of Pediatrics, University of Minnesota | |
| 2:50 pm - 3:10 pm | Hermitage E/F | Genetic Barcoding for Improving Gene-Therapy Research in Sanfilippo Syndrome IIIB Research [More Info] Ricky Ashby, MPH, MD-PhD Graduate Student, University of Florida MD-PhD Training Program, University of Florida College of Medicine | |
| 3:10 pm - 3:20 pm | Hermitage E/F | Question and Answer Panel | |
| 3:20 pm - 4:10 pm | Hermitage E/F | Session IV: Society Funded Research Updates [More Info] N. Matthew Ellinwood, DVM, PhD, Chief Scientific Officer, National MPS Society Mark Sands, PhD, Professor in the Departments of Medicine and Genetics, Washington University School of Medicine | |
| 3:20 pm - 3:40 pm | Hermitage E/F | Developing MRI to Distinguish Neuropathic and Nonneuropathic Forms of MPS II [More Info] Igor Nestrasil, MD, PhD, Research Assistant Professor, Department of Pediatrics, University of Minnesota | |
| 3:40 pm - 4:00 pm | Hermitage E/F | Imaging Cells to Tell Which IDS Mutations Predict Hunter Syndrome Phenotypes [More Info] William Buchser, PhD, Assistant Professor, Department of Genetics; Director of FIVE@MGI, Washington University in St. Louis School of Medicine | |
| 4:00 pm - 4:10 pm | Hermitage E/F | Question and Answer Panel | |
| 4:10 pm - 4:30 pm | Hermitage E/F | Scientific Sessions Recap and Open Question Forum | |
| 1:50 pm - 2:10 pm | Hermitage Lobby | Health Break | |
| Various Locations | Family Conference, Saturday 1:00 - 5:00 pm | ||
| 08/06/2022 | |||
| 1:00 pm - 3:00 pm | Various Locations | Session V - Family Conference 1:00 - 4:00 pm Concurrent Sessions | |
| 1:00 pm - 2:00 pm | Oaklands | Medical Trauma in Patients and Families [More Info] Melissa Hogan, JD, MPS II Parent, Founder of Project Alive | |
| 1:00 pm - 2:00 pm | Belle Meade | Genetic Testing/Family Planning [More Info] Barbara Burton, MD, Professor of Pediatrics - Genetics, Birth Defects, and Metabolism, Northwestern University | |
| 1:00 pm - 2:00 pm | Evergreen | Navigating IEPs and Educational Needs [More Info] Leslie Urdaneta, MSW, LCSW, Director of Family Support & Communication, National MPS Society | |
| 1:00 pm - 2:00 pm | Various Locations | (Concurrent Sessions - Please Choose) | |
| 2:00 pm - 3:00 pm | Oaklands | Medical Trauma in Patients and Families (Recurring) [More Info] Melissa Hogan, JD, MPS II Parent, Founder of Project Alive | |
| 2:00 pm - 3:00 pm | Evergreen | Navigating IEPs and Educational Needs (Recurring) [More Info] Leslie Urdaneta, MSW, LCSW, Director of Family Support & Communication, National MPS Society | |
| 2:00 pm - 3:00 pm | Belle Meade | Genetic Testing/Family Planning (Recurring) [More Info] Barbara Burton, MD, Professor of Pediatrics - Genetics, Birth Defects, and Metabolism, Northwestern University | |
| 4:00 pm - 5:00 pm | Belle Meade | Session VI: You Matter Too! Health and Wellness [More Info] Carla S. Jackson, LCSW, Pediatric Social Worker Palliative Care and Complex Care Programs , Monroe Carell Jr. Children’s Hospital at Vanderbilt Cortni Dubuc, LMSW, Hematology/Oncology Pediatric Social Worker , Monroe Carell Jr. Children’s Hospital at Vanderbilt | |
| 5:00 pm - 5:30 pm | Belle Meade | Remembrance Ceremony | |
| 6:30 pm - 8:30 pm | Hermitage C/D/E | National MPS Society Banquet and Talent Show | |
| 8:30 pm - 10:30 pm | Hermitage F | Nashville Party with onsite music and beverages | |
| 8:45 pm - 10:45 pm | Hermitage A/B | Camp Courage Opens for Nashville Party | |
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