Agenda at a Glance

 

Wednesday, August 1 Session Speaker(s) Location
1:00pm – 5:00pm  International Network Meeting –  Track 1
*Invitation only*
(Break Included)
TBD
12:15pm Screening Weight of the Mountain Michelle Berg, Abeona
1:00 pm
2:00 pm

Sanfilippo, LSD Manifestation in the CNS
Living the Best Quality Life – Pharmacology for MPS III
Break
Maria Escolar, MD, MS
Jeanine Jarnes, PharmaD
3:15 pm

4:00 pm

Industry Updates

Therapeutic Approaches for Sanfilippo Syndrome
Michelle Berg, Samantha Parker
Mark Rothera
Patricia Dickson, MD
5:00pm – 8:00pm
Registration Maritime Foyer
6:00pm – 9:00pm
International MPS Network Dinner
Old Town- 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
3:15pm – 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
United Kingdom
Grande Ballroom A
5:00pm – 5:30pm
Break and Exhibitor Session   Harbor Island 2 & 3
5:30pm – 6:00pm
21st Century Key Insights and Screening for Rare Diseases

Kim Angel, Moderator
Canada MPS Society
Grande Ballroom A 
5:30pm – 6:00pm Key Insights Into Disease Pathogenesis 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  Alfred Wiesbauer, MPH
Moderator, Switzerland
 Grande Ballroom A
 8:00am – 8:20am  Measuring Neurocognitive Function:Best Practices in Clinical Management and Clinical Trials
Elsa Shapiro, PhD, ABPP
United States
 Grande Ballroom A
8:20am – 8:40am The Impact of Neurobehavioral Symptoms from Clinical Trials Through Adulthood
Julie Eisengart, LP, PhD
United States
8:40am – 9:00am Connecting the Dots: The Role of Caregiver Preference in Drug Development Cara O’Neill, MD
United States

 Grande Ballroom A
9:10am – 9:20am Rare Disease Drug Development: Challenges and Opportunities Through the Regulatory Lens
Patroula Smpokou, MD FACMG
FDA, United States
Grande Ballroom A
9:20am – 9:30am Question and Answer Session Panel Presenters Grande Ballroom A
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
Stephanie Bozarth, MSW
Moderator United States
 
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
United Kingdom
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  Bob Stevens, Group CEO
Moderator, United Kingdom

 
1:00pm – 1:30pm Publishing Soon Michael Beck, MD
Germany
1:30pm – 2:00pm
Expressions: Gene Therapy 101 and Gene Editing Overview Mark Sands, PhD
United States
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 – Transitioning to Adults
My Life is More than My Diagnosis

 Terri Klein, NPGC
Moderator, United States
Grande Ballroom A
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
Grande Ballroom A
4:30pm – 4:40pm I Don’t Belong in a Bubble! Sheri Wise, MPS IVA Grande Ballroom A
4:40pm – 4:50pm Knowledge is Power, Mastering Life with a Rare Disease Jennifer Klein, ML III Grande Ballroom A
4:50pm – 5:00pm Making Lemonade Kendra Gottsleben, MPS VI Grande Ballroom A
5:00pm – 5:10pm Orthopedic Challenges Adele, Kary, MPS I Grande Ballroom A
5:10pm – 5:20pm
Conquering Adult Transitions
Erica Thiel, MPS I
Grande Ballroom A 
5:20pm – 5:30pm
My Life is More Than My Diagnosis-
Learning to Live Well with MPS Q&A
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
Terri Klein, NPGC
Moderator, United States
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
Michelle Berg
Grande Ballroom A
8:10am – 8:20am Armagen, MPS I Therapy Mathias Schmidt, PhD
Grande Ballroom A
8:20am – 8:30am Regenxbio, MPS I and MPS II Stephen Yoo, M.D.
Grande Ballroom A
8:30am – 8:45am Industry Q&A Panelist Q&A Grande Ballroom A
8:45am – 9:05am
Reviewing Today’s Science and Complex Therapeutic Approaches Lorne Clarke, MD, CM, FRCPC, FCCMG – Canada
Grande Ballroom A
9:05am – 9:15am Navigating Clinical Trials with a Family – How One Family Unit Makes Decisions, the New Frontier Steve Holland, Pres. Emer.
United States
Grande Ballroom A
9:15am – 9:45am Managing and Complex Care Management in the Patient – Collaborative Efforts
Kendra Bjoraker, PhD
United States
Grande Ballroom A
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
Jenny Noble, Moderator
ISMRD
Grande Ballroom A
10:00am – 10:20am
Alpha Mannosidosis- Enzyme Replacement Therapy Updates Paul Harmatz, MD
United States
Grande Ballroom A
 10:20am – 10:40am
Mucolipidosis – Gene Therapy
Science Updates
Steven Gray, PhD
United States
Grande Ballroom A
 10:40am – 11:20am
Understanding the Pain, Anxiety, and Depression and Chronic Conditions; MPS and Related Diseases
Kendra Bjoraker, PhD
United States
Grande Ballroom A
11:20am – 11:40pm
Managing the Complex Journey for MPS II
Melissa Hogan, JD
United States
Grande Ballroom A
11:40am – 11:55pm Managing Later Onset MPS III Symptoms Carl Kapes
United States
Grande Ballroom A
  12:00pm – 1:00pm
 Lunch   Pavilion
1:00pm – 3:00pm
Charting Optional Therapies / Research Animal Model Updates Michaela Weigl, Moderator
Austria
Grande Ballroom A
1:00pm – 1:45pm HCST- The Road Today – Protocols and Outcomes Paul Orchard, MD
United States
Grande Ballroom A
1:45pm – 2:00pm
Bisphosphonates Presentation
Jenny Noble
New Zealand
Grande Ballroom A
2:00pm – 2:15pm Efficacy of TKIs to reduce Heperan Sulfate via Autophagy Alan Fowler
United States
2:15pm – 2:45pm
Morquio Updates- Surgical/Trachea Care Shunji Tomatsu, MD, PhD
United States
Grande Ballroom A
3:00pm –  4:00pm
Writing our Future for MPS and Related Diseases,
Ultimate Advocacy and 2020 Presentation

Carmen Kunkel, Moderator Germany
Grande Ballroom A
3:00pm –  3:30pm
Collaborative Efforts for Family Caregivers – Create your Talk Terri Klein, NPGC
United States
Grande Ballroom A
3:30pm – 3:45pm
Registries, Advocacy, Newborn Screening – Magnifying the Patient Voice
Stephanie Bozarth, MSW
United States
Bob Stevens, Group CEO
United Kingdom
Grande Ballroom A
3:45pm – 4:00pm 2020 International Symposium Presentation – Barcelona, Spain
Ana Mendoza, President Spanish MPS Society and
Jordi Cruz, Executive Spain
Grande Ballroom A
4:00pm – 5:00pm
Remembrance Ceremony    Bayview Lawn
6:30pm
Banquet Dinner and Awards   Grande Ballroom A