Agenda at a Glance

 

Wednesday, August 1 Session Speaker(s) Location
1:00pm – 5:00pm  International Network Meeting –  Track 1
*Invitation only*
(Break Included)
Executive Center 1
1:00pm – 5:00pm  Sanfilippo Meet-up
Marina 6
5:00pm – 8:00pm
Registration Maritime Foyer
6:00pm – 9:00pm
International MPS Network Dinner
Old Towne- Offsite
Thursday, August 2 Session  Speaker(s)   Location
7:00am – 5:00pm
Registration   Maritime Foyer
7:00am – 8:00am International Network Breakfast
*Invitation only*
  Executive Center 1
7:30am – 2:00pm Optional Event($50) San Diego Zoo
(includes breakfast and lunch)
  Offsite
8:00am – 12:00pm
International Network Meeting – Track 2 Morning   Executive Center 1
12:00pm – 1:00pm
International Network Meeting Lunch   Executive Center 1
1:00pm – 3:00pm
International Network Meeting – Track 2 Afternoon   Executive Center 1
3:00pm – 7:00pm
Exhibitor Hall Hours   Harbor Island 2 & 3
3:00pm – 3:30pm
Break and Exhibitor Session   Harbor Island 2 & 3
3:15pm – 4:00pm
Opening Plenary and Welcoming Address Patti Dickson, MD and Stephanie Bozarth, MSW
United States  
Grande Ballroom A
4:00pm – 4:10pm  21st Century Rare Disease Ecosystem – Research, Science, Regulatory Patient Centered Care Video Welcome
Emil Kakkis, MD, PhD
United States 
Grande Ballroom A
4:10pm – 4:20pm  MPS Adult Keynote Presenter Melissa Bilodeau, MPS IVA
Canada
Grande Ballroom A
4:20pm – 4:30pm MPS Adult Keynote Presenter  Carolina Hartman, MPS VI
Brazil
Grande Ballroom A
4:30pm – 5:10pm Scientific Challenges of New Therapies in the 21st Century Brian Bigger, PhD
UK 
Grande Ballroom A
5:00pm – 5:30pm
Break and Exhibitor Session   Harbor Island 2 & 3
5:30pm – 6:00pm
Key Insights Into Disease Pathogenesis
Importance for MPS Societies Involvement
Patient Centered Care 
Joseph Muenzer, MD, PhD
United States
Grande Ballroom A 
6:00pm – 6:30pm
Newborn Screening, Collapsing the Diagnostic Odyssey
for Future Generations
Barbara Burton, MD
United States
Grande Ballroom A
6:30pm – 7:00pm
Diagnostic Journey, Unlocking the Mystery
for Rare Diseases
Jama Nateqi, PhD
Austria
Grande Ballroom A
7:00pm  to 9:00pm
Welcome Reception
  Bayview Lawn
Friday, August 3 Session  Speaker(s)  Location 
 7:00am – 8:00am
Breakfast   Pavilion
7:00am – 6:00pm
Registration   Maritime Foyer
8:00am – 9:30am Opening Session Clinical Management I Presentations  Elsa Shapiro, PhD, ABPP
United States 
 Grande Ballroom A
 8:00am – 8:30am  Neurological Manifestations of MPS Elsa Shapiro, PhD, ABPP
United States
 Grande Ballroom A
8:30am – 8:50am Clinical Trial Designs in MPS;
Understanding Endpoints
Cara O’Neill, MD
8:50am – 9:10am Behavioral Manifestations of MPS-
Successfully Identifying Obstacles for Clinical Trials 
Julie Eisengart, LP, PhD
United States
 Grande Ballroom A
9:10am – 9:30am Understanding Regulatory Strategies in Clinical Trial Design Publishing Soon
9:00am – 5:00pm
Exhibit Hall Hours   Harbor Island 2 & 3
 9:30am – 10:00am
 Break and Exhibitor Session    Harbor Island 2 & 3
 10:00am – 12:00pm
Clinical Management II –
Cardiac and Bone  Disease
   
10:00am – 10:15am
Overview of Musculoskeletal Disease in MPS and ML
(Q&A 5 min)
Klane White, MD, MSc
United States
Grande Ballroom A
10:15am – 10:30am
Hip Disease in MPS (Q&A 5 min)
Kevin Walker, MD
United States
Grande Ballroom A
10:30am – 10:45am Treatment of Lower Extremities Alignment in MPS  (Q&A 5 min) Deborah Eastwood, FRCS, MB
UK
Grande Ballroom A
10:45am – 11:00am Spinal Pathology in MPS  (Q&A 5 min) Klane White, MD, MSc
Grande Ballroom A
11:00am – 11:10am Hip Dysplasia Debate – Panel One Orthopedic Panel
Grande Ballroom A
11:10am – 11:20am Thoracolumbar Gibbous Debate – Panel Two Orthopedic Panel
Grande Ballroom A
11:20am – 11:35am Orthopedic Panel and Q&A Orthopedic Panel
Grande Ballroom A
11:35am – 11:55am
Cardiac Manifestations Across
MPS and Related Diseases
Raymond Wang, MD
United States
Grande Ballroom A
 12:00pm – 1:00pm
Lunch    Pavilion
 1:00pm – 1:30pm Management II- Next Generation Treatments/Combination Therapies and Advancing Clinical Trials  Michael Beck, MD
Germany
 
1:30pm – 2:00pm
Expressions: Gene Therapy 101 and Gene Editing Overview Mark Sands, PhD, USA
Grande Ballroom A
2:00pm – 2:30pm
Gene Therapy and Clinical Trials Gene Delivery IT/IV Chet Whitley, MD, PhD, USA
Grande Ballroom A
 2:30pm – 3:00pm  Break and Exhibitor Session     Harbor Island 2 & 3
3:00pm – 3:30pm  Scientific Challenges of New Therapies in MPS/Related Diseases and Stem Cell Gene Therapy Updates for MPS II and MPS III A/B Brian Bigger, PhD, UK
Grande Ballroom A
3:30pm – 4:00pm Clinical Presentations, Managing Immunological
Obstacles in Patients
Paul Harmatz, MD
United States
Grande Ballroom A
4:00pm – 5:30pm
Management II- Optional Therapies, Adult MPS Track
 
4:00pm – 4:30pm Conquering Management of MPS and Related Diseases As an Adult, Transitioning Pediatric to Adult Care Stacy Hewson, MS, MSc, CCGC, CGC, Canada
4:30pm – 4:45pm
Adult Presentations
Publishing Soon Grande Ballroom A 
4:45pm – 5:25pm
My Life is More Than My Diagnosis-
Learning to Live Well with MPS
MPS/ML Adult Panel Grande Ballroom A
 5:30pm – 9:00pm Adult Boat Cruise for those with MPS
and related diseases
MPS/ML Adults  Offsite
Saturday, August 4th  Session  Speaker(s)  Location 
 7:00am – 8:00am Breakfast   Pavilion
7:00am – 5:00pm Registration   Maritime Foyer
 8:00am – 9:30am
Importance of Research and
Advocacy Group Collaboration
Michael Beck, MD
Germany
Grande Ballroom A
Emerging Therapies in Frontier Science – Panel of Industry Entreprenuership/Industry Development in Rare Diseases
 8:00am – 8:10am
Abeona, MPS IIIA/ MPS IIIB Gene Therapy Tim Miller, PhD
United States
Grande Ballroom A
8:10am – 8:20am Armagen, MPS I Therapy Mathias Schmidt, PhD
United States
Grande Ballroom A
8:20am – 8:30am  Regenxbio, MPS I and MPS II Therapy Coming Soon
8:30am – 8:45am Industry Q&A Panelist Q&A
8:45am – 9:15am
Ethical Guidelines for Advocacy Groups
and Industry to Collaborate
Anya Prince, MPP, JD
United States
Grande Ballroom A
9:15am – 9:45am Optional Therapies- How Do Families Make Decisions Publishing Soon
9:00am – 3:00pm Exhibit Hall Hours   Harbor Island 2 & 3
9:30am – 10:00am  Break and Exhibitor Session   Harbor Island 2 & 3
10:00am – 12:00pm Charting New Science
in MPS and Related Diseases- the Unmet Need
Chester Whitley, MD, PhD
United States
Grande Ballroom A
10:00am – 10:20am
Alpha Mannosidosis- Enzyme Replacement Therapy Updates Publishing soon
Grande Ballroom A
 10:20am – 10:40am
Mucolipidosis – Gene Therapy
Science Updates
Steven Gray, PhD
United States
Grande Ballroom A
 10:40am – 11:20am
Managing Pain and Anxiety of a Rare Disease
in the Absence of Therapy
Terri Klein, NPGC
United States
Grande Ballroom A
11:20am – 12:00pm
Later Stage Care for MPS II and MPS III Publishing soon Grande Ballroom A
  12:00pm – 1:00pm
 Lunch   Pavilion
1:00pm – 3:00pm
Charting Optional Therapies / Research Animal Model Updates Terri Klein, NPGC
United States
Grande Ballroom A
1:00pm – 1:45pm HCST- The Road Today – Protocols and Outcomes Paul Orchard, MD
United States
Grande Ballroom A
1:45pm – 2:15pm
Cornea Transplants Publishing soon Grande Ballroom A
2:15pm – 2:45pm
Morquio Updates- Surgical/Trachea Care Shunji Tomatsu, MD, PhD
United States
Grande Ballroom A
2:45pm –  3:00pm
Writing our Future for MPS and Related Diseases, Ultimate Advocacy and 2020 Presentation Lorne Clarke, MD
Canada 
Grande Ballroom A
3:00pm –  3:30pm
Registries, Advocacy, Newborn Screening- Magnifying the Patient Voice  Stephanie Bozarth, MSW,
United States
Bob Stevens,
UK
Grande Ballroom A
3:30pm – 4:00pm
Effective Collaboration- Strength in Numbers Publishing soon Grande Ballroom A
3:45pm – 4:00pm 2020 International Symposium Presentation Publishing soon Grande Ballroom A
4:00pm – 5:00pm
Remembrance Ceremony    Bayview Lawn
6:30pm
Banquet Dinner and Awards   Grande Ballroom A