Schedule

THURSDAY MAY 19

Morning

1000-1200

CIMDRN Meeting

Lunch

1200-1300 – Lunch Symposium

Home Infusion Therapy for Patients living with Mucopolysaccharidosis Type I (MPSI) in Canada | Sponsored by Genzyme

Afternoon

1300-1400

A New Paradigm for Metabolic Medicine

 

1400-1600 – Concurrent Sessions

 

Medunik Advisory Board Meeting

 

Dietitians’ Meeting/Webinar:

  • Dietary Practices of Inherited Metabolic Disorders in the UK
  • Recent Clinical Data and Experience with GMP in PKU Treatment
  • Experiencing the PKU Diet, an RD’s Perspective

Highlights and Updates on Fabry Disease: Adult and Pediatric Outcomes

 

1405-1425

Fabry Disease in Children. Expectant Follow Up or ERT?

 

1425-1455

Management and Outcomes in Adults with Fabry Disease – Lessons From the CFDI Study

 

1455-1525

Fabry Disease: Who to Treat and When to Stop. The VUS Project and the Canadian Fabry Treatment Guidelines

 

1525-1540

When Enzyme Replacement Therapy Fails – Case Presentation

 

1540-1600

Discussion

 

1600-1700
Treatment Considerations for Neurodegenerative IEMs: Leukodystrophies and Epilepsies

 

1700-1800
Fatty Acid Oxidation PKU

Evening

1830-2100

Welcome Reception

FRIDAY MAY 20

Breakfast

0730-0830 – Breakfast Symposium

A Difficult to Identify Metabolic Disease: Late Onset Lysosomal Acid Lipase Deficiency | Sponsored by Alexion

Morning

0830-0835

Welcome Remarks

 

0835-1000

Morning Session 1: The National Working Group Experience
Canadian Inherited Metabolic Diseases Research Network (CIMDRN)

 

1000-1030
Refreshment Break & Poster Viewing

 

1030-1230

Morning Session 2: Development of Treatments, Evidence and Policy

 

1030-1100
Introduction/Overview: A 360 Degree Review on Rare Disease Drug Development

 

1100-1130
Development of Treatments, Evidence and Policy

 

1130-1200
Platform Presentations of Selected Abstracts

1200–1230
Assessment of Health Technologies and Value Based Decision Making

 

Lunch

1230-1330 – Lunch Symposium

New Evidence and Treatment in the Management of PKU | Sponsored by BioMarin

Afternoon

1330-1445

Afternoon Session 1: Mitochondrial Disorders and Innovative Therapies 1

 

1330-1345

MitoCanada

 

1345-1400

Platform Presentation of Selected Abstracts

1400-1445

Treatment of Mitochondrial Disorders

 

1445-1500
Refreshment Break & Poster Viewing

 

1500-1730

Afternoon Session 2: Innovative Therapies 2

 

1500-1545

Cell Based Therapies for Metabolic Disorders

 

1545-1630

Treatment of the Hepatic Glycogen Storage Diseases

 

1630–1730

Updates and Evidence: Management and Treatment of Urea Cycle Disorders

 

1730-1830

Cocktails and Poster Viewing

Evening

1900-2200

Garrod Gala
Lobster Dinner with entertainment at Pier 21 Museum

Garrod Pioneer Award Presentation

SATURDAY MAY 21

Breakfast

0800-0900

Breakfast Symposium | Patient Directed Outcomes

Morning

0900-1100

Morning Session 1: High Throughput Technologies for Personalized Management of IEM

 

0900-0930

Genomics to Diagnose Neurometabolic Disease: Bigger Always Better?

 

0930-1000

Expanding Screening and Diagnostic Capacity for Amino Acidurias Through Tandem Mass Spectrometry

 

1000-1030

New Twists on Old Biomarkers

 

1030-1100

Mass Spectrometry Biomarker Analysis for the Identification, Monitoring and Follow-up of Patients

 

1100-1115
Refreshment Break & Poster Viewing

 

1115-1300

Morning Session 2: Enhancement of Established Therapies

 

1115-1215

Updates on Guidelines for Dietary Treatment of IEM

 

1115-1145

European Dietary Guidelines of Propionic Acidaemia (PA) and Methylmalonic Aciduria (MMA)

 

1145-1215

Dietary Management of Tyrosinemia

 

1215-1245

Platform Presentations of Selected Abstracts

1245-1300

Wrap-up and Closing Remarks

Lunch

1300-1400

Principles in Biomarker Discover and Translational Research in Lysosomal Storage Diseases | Sponsored by Shire

Afternoon

1400-1600
Garrod Association Membership Meeting

 

1700
Closing Social – Brewery Tour and Unwind!

SUNDAY MAY 22

 

Annual CanPKU Meeting
Location: IWK Health Centre, Halifax NS