36th Annual Family Conference Nashville 2022
 
 
 
08/04/2022
7:30 am - 12:00 pmBoard of Directors Meeting
2:00 pm - 8:00 pmRegistration
2:45 pm - 9:45 pmCamp Courage Opens
3:00 pm - 3:05 pmWelcome Announcements
3:00 pm - 3:30 pmOpening Keynote Speaker - Mark Sands, PhD
3:00 pm - 10:00 pmReflection Area
3:30 pm - 4:00 pmScience Leading to New Therapies for MPS
4:20 pm - 4:50 pmNewborn Screening Updates
4:50 pm - 5:15 pmMPS I and MPS II Newborn Screening State by State Advocacy
5:00 pm - 8:00 pmMPS Music City Run, Rock and Roll 5K Package Pick-Up
5:15 pm - 6:00 pmFoundations Roundtable - Collaborations to Help the Patient Community
6:00 pm - 7:30 pmWelcome Buffet Dinner
6:00 pm - 7:30 pmNewly Diagnosed Family Dinner (diagnosed since December 2019) Overview and Coping with MPS and ML
7:30 pm - 8:30 pmMPS I - By Syndrome Breakout Session
7:30 pm - 8:30 pmMPS II - By Syndrome Breakout Session
7:30 pm - 8:30 pmMPS III - By Syndrome Breakout Session
7:30 pm - 8:30 pmMPS IV - By Syndrome Breakout Session
7:30 pm - 8:30 pmMPS VI - By Syndrome Breakout Session
7:30 pm - 8:30 pmMPS VII - By Syndrome Breakout Session
7:30 pm - 8:30 pmML II/III - By Syndrome Breakout Session - Mercedes Ramirez Johnson, Moderator
7:30 pm - 9:30 pmSibling Breakout Sessions
8:30 pm - 9:30 pmFamily Breakout: Mothers
8:30 pm - 9:30 pmFamily Breakout: Fathers
8:30 pm - 9:30 pmFamily Breakout: Grandparents
8:30 pm - 9:30 pmFamily Breakout: Adults with MPS/ML
8:30 pm - 9:30 pmFamily Breakouts: Bereaved Families
9:45 pmCamp Courage Closes
 
08/05/2022
6:30 am - 8:00 amBreakfast Buffet
7:45 amCamp Courage Opens
7:45 am - 12:00 pmSession I - MPS and ML Management and Care
7:45 am - 8:00 am2022 Fundraiser Award - Kimber Heiling, MPS VI Mother
7:45 am - 8:00 am2022 Chairman Award - Rev. Wayne Eppehimer
8:00 am - 8:45 amOverview and Treatment Status of MPS and ML II/III
8:45 am - 9:30 amOrthopedic Updates in MPS
9:30 am - 10:00 amAnesthesia and Airway Complications
10:00 am - 10:30 amBlood Stem Cell Transplantation & Gene Therapy for Hurler Syndrome
10:40 am - 11:00 amQ & A Session
11:00 am - 11:45 amFunctional outcomes in MPS: Neurocognitive Function and Beyond
11:45 am - 1:00 pmLunch Buffet
12:15 pmCamp Courage Closes
12:45 pmCamp Courage Opens
12:45 pm - 2:45 pmSession II - Patient and Family Warriors
12:45 pm - 1:05 pm2022 Directors' Award - Christine Tippett, MPS IV Mother, Board of Directors
12:45 pm - 1:05 pmVisionary Leadership Award - Dr. Elizabeth F. Neufeld, PhD
1:00 pm - 1:45 pmAdults with MPS or ML Panel
1:45 pm - 2:15 pmMission Fundraising - Get Involved
2:15 pm - 2:45 pmSiblings of MPS and ML Patients
2:45 pm - 3:00 pmHealth Break
Session III - Industry and Clinical Trial Updates
3:00 pm - 3:15 pmMPS Family Conference:Takeda Update
3:15 pm - 3:30 pmJCR Presents Latest Updates on Pabinafusp alfa, JR-171, JR-441, and JR-446
3:30 pm - 3:45 pmREGENXBIO Interim Analysis of Data From the RGX-121 Gene Therapy Clinical Trial for the Treatment of Severe MPS II
3:45 pm - 4:00 pmSanofi presents: All About MPSI Disease; Information for Patients, Families and Friends
4:00 pm - 4:15 pmHomology New One-Time Approach to Treating MPS II: HMI-203 Investigational Gene Therapy
4:15 pm - 4:30 pmUltragenyx MPS Updates
4:30 pm - 4:45 pmDenali Therapeutics Update on DNL 310 Clinical Program
4:45 pm - 5:00 pmOrchard Therapeutics Presents Hematopoietic Stem Cell Gene Therapy
5:00 pm - 5:15 pmBioMarin's Partnership with the MPS Community
5:45 pmCamp Courage Closes
6:30 pmNashville Night on Your Own
 
08/06/2022
6:30 am - 11:30 amNational MPS Society Walk and Roll 5K Run and 1 Mile Walk
6:30 am - 7:45 amGrab and Go Breakfast and Board Buses
8:00 am - 11:00 am5K Chip Timed Run
8:00 am - 11:00 amFamily Walk
8:00 am - 11:00 amCelebration Lunch
10:45 am - 11:30 amBoard Buses Back to Sheraton; Return to Conference Sessions
 
 
Scientific Conference, Saturday Noon - 4:30 pm
 
08/06/2022
12:00 pm - 12:10 pmWelcome, Overview, and New Developments in Science of MPS and at the Society
12:10 pm - 1:00 pmSession I: Society Funded Research Updates
12:10 pm - 12:30 pmA Clinically Relevant MPS IVA Animal Model
12:30 pm - 12:50 pmEarly Development of MPS IIIA Dopaminergic Neurons: At the Nexus of Behavior Changes and Therapy
12:50 pm - 1:00 pmQuestion and Answer Panel
1:00 pm - 1:50 pmSession II: Society Funded Research Updates
1:00 pm - 1:20 pmRetracing the Developmental Roots of MPS II Neuropathology
1:20 pm - 1:40 pmAutoimmune Component of Sanfilippo Syndrome
1:40 pm - 1:50 pmQuestion and Answer Panel
2:10 pm - 3:20 pmSession III: Fellow Research in the MPSs
2:10 pm - 2:30 pmPreliminary Examination of Oligodendrocytes in the MPS VII Mouse Model
2:30 pm - 2:50 pmCognitive Function in MPS Phenotypes That Do Not Have Decline
2:50 pm - 3:10 pmGenetic Barcoding for Improving Gene-Therapy Research in Sanfilippo Syndrome IIIB Research
3:10 pm - 3:20 pmQuestion and Answer Panel
3:20 pm - 4:10 pmSession IV: Society Funded Research Updates
3:20 pm - 3:40 pmDeveloping MRI to Distinguish Neuropathic and Nonneuropathic Forms of MPS II
3:40 pm - 4:00 pmImaging Cells to Tell Which IDS Mutations Predict Hunter Syndrome Phenotypes
4:00 pm - 4:10 pmQuestion and Answer Panel
4:10 pm - 4:30 pmScientific Sessions Recap and Open Question Forum
1:50 pm - 2:10 pmHealth Break
 
 
Family Conference, Saturday 1:00 - 5:00 pm
 
08/06/2022
1:00 pm - 3:00 pmSession V - Family Conference 1:00 - 4:00 pm Concurrent Sessions
1:00 pm - 2:00 pmMedical Trauma in Patients and Families
1:00 pm - 2:00 pmGenetic Testing/Family Planning
1:00 pm - 2:00 pmNavigating IEPs and Educational Needs
1:00 pm - 2:00 pm(Concurrent Sessions - Please Choose)
2:00 pm - 3:00 pmMedical Trauma in Patients and Families (Recurring)
2:00 pm - 3:00 pmNavigating IEPs and Educational Needs (Recurring)
2:00 pm - 3:00 pmGenetic Testing/Family Planning (Recurring)
4:00 pm - 5:00 pmSession VI: You Matter Too! Health and Wellness
5:00 pm - 5:30 pmRemembrance Ceremony
6:30 pm - 8:30 pmNational MPS Society Banquet and Talent Show
8:30 pm - 10:30 pmNashville Party with onsite music and beverages
8:45 pm - 10:45 pmCamp Courage Opens for Nashville Party