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*
  TBD
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   TBD
12:00pm – 1:00pm
International Network Meeting Lunch   TBD
1:00pm – 3:00pm
International Network Meeting – Track 2 Afternoon   TBD
3:00pm – 7:00pm
Exhibitor Hall Hours   Harbor Island 2 & 3
3:00pm – 3:30pm
Break and Exhibitor Session   Harbor Island 2 & 3
3:30pm – 4:00pm
Opening Plenary and Welcoming Address Patti Dickson, MD and Stephanie Bozarth, MSW
United States  
Grande Ballroom A
3:30pm – 3:45 pm  21st Century Rare Disease Ecosystem – Research, Science, Regulatory Patient Centered Care Video Keynote:
Emil Kakkis, MD, PhD
United States 
Grande Ballroom A
3:45pm – 4 pm  MPS Adult Keynote Presentations Melissa Bilodeau, MPS IVA
Canada
Grande Ballroom A
4:15pm – 4:30 pm MPS Adult Keynote Presentations Sura Hane, MPS VI
Switzerland
Grande Ballroom A
4:30pm – 5:00 pm 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 – 9:00am Clinical Trial Designs in Lysosomal Diseases;
Understanding Endpoints
Publishing soon
9:00am – 9:20am Behavioral Manifestations of MPS-
Successfully Identifying Obstacles for Clinical Trials 
Julie Eisengart, LP, PhD
United States
 Grande Ballroom A
9:20am – 9:45am 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 – 10:30am
Bone Disease and Orthopedic Panel
Klane White, MD, MSc
United States
Grande Ballroom A
10:30am – 10:45am
Hip Replacement and Reconstruction Outcomes in MPS –
New Technologies
Kevin Walker, MD
United States
Grande Ballroom A
10:45am – 11:00am Lower Extremities Alignment Outcomes in MPS Deborah Eastwood, FRCS, MB
UK
Grande Ballroom A
11:00am – 11:15am Spinal Pathology in MPS Publishing soon
Grande Ballroom A
11:15am – 11:30 am Orthopedic Panel and Q&A All presenters Grande Ballroom A
11:30am – 11:50am
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 Olivier Danos, PhD
United States
Grande Ballroom A
2:00pm – 2:30pm
Pre-clinical Overview Speakers publishing soon Grande Ballroom A
 2:30pm – 3:00pm  Break and Exhibitor Session     Harbor Island 2 & 3
3:00pm – 3:30pm  Gene Therapy and Clinical Trials:
Gene Delivery Differentials IT/IV
Chet Whitley, Md, PhD
United States
Grande Ballroom A
3:30pm – 4:00pm Clinical Presentations, Managing Immunoligical
Obstacles in Patients
Paul Harmatz, MD
United States
Grande Ballroom A
4:00pm – 5:30pm
Management II- Gene Therapies, Adult MPS Track
 
4:00pm – 4:15pm Gene Therapy Clinical Trials Expectations/Preparedness
(Patient Perspective)
 Publishing soon 
4:15pm – 4:45pm
Conquering Management of MPS and Related Diseases
As An Adult, Transitioning Pediatric to Adult Care 
Stacy Hewson, MS, MSc, CCGC, CGC
Canada
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 Sangamo Therapeutics
 TBD
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 and Melissa Hogan,
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