Welcome to the Garrod Association

Contact Us: Please email GarrodAssociation@outlook.com with your questions or comments so they may be directed to the appropriate person.  Thank you.

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Open Call for Metabolic Disorder Research Catalyst Proposals – Deadline Extended to May 3, 2019

The RDMM is partnering again with the FGDPL for an open call – please note that the deadline has been extended to May 3, 2019. Please click here for additional information. 

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The 2019 GARROD Symposium will be held from May 9 – 11, 2019 at The Sheraton Centre.

If you wish to get on a mailing list to receive updates for this symposium, please contact jo.ipce@ubc.ca

Registration and Brochure are available – links below

For registration details, please click here.

Click here to view the program.

Location and Accommodation
The Sheraton Centre, Toronto, ON
123 Queen Street West
Toronto, Ontario M5H 2M9

Details on the room block and rates can be found  here.

Satellite Symposiums

Please see details and information below. 

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Horizon Therapeutics Canada – May 9, 9:30-11:30 AM –

UCD Diagnosis, Management & Treatment

*Breakfast will be provided

Presenter: Dr. Barbara Burton
Professor of Pediatrics at the Northwestern University Feinberg School of Medicine,
Clinical Practice Director; Division of Genetics, Birth Defects and Metabolism at the Ann & Robert H. Lurie Children’s Hospital of Chicago

Objectives

  1. Discuss current diagnosis, management & treatment of Urea Cycle Disorders
  2. Understand the risks associated with high ammonia levels
  3. Provide the latest clinical data for RAVICTI® (glycerol phenylbutyrate) Oral Liquid
  4. Discussion & Feedback Session

You can view the Save the Date poster here.

Please register for the Symposia here – click on link below:
https://www.antibodyevents.com/2019UCDSymposium

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Recordati Rare Diseases Canada – May 9, 2:00-3:30 PM –

Management of propionic, methylmalonic and isovaleric acidurias in Canada

  1. Saadet Andrews (30 minutes): Canadian Metabolic Physician practices in the acute and chronic management of PA, MMA and IVA
  2. Chitra Prasad (30 minutes): International treatment guidelines for PA, MMA and IVA
  3. Discussion (30 minutes)

Objectives

  1. Recognize the phenotypes of PA, MMA and IVA in Canada
  2. Summarize Canadian physician practices for the management of PA, MMA and IVA
  3. Review international treatment guidelines of PA, MMA and IVA

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An International Registry for Morquio-B Disease hosted at BC Children’s Hospital

Morquio-B-Disease is an ultra-rare form of late-onset GM1-Gangliosidosis, characterized by bone disease resembling Morquio-A Disease.

Various research consortia are currently developing treatments for this condition.

Funding from the Priest Family Foundation (www.morquiob.com) allows us to collect natural history data, which will be most essential for the future design of clinical trials.

If you have patients with Morquio-B-Disease or with late-onset GM1-Gangliosidosis, please contact us.

Every single patient is important.

Sylvia Stockler (PI) sstockler@cw.bc.ca

Maria Boldut (Project Manager) maria.boldut@cw.bc.ca

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Rare Diseases: Models & Mechanisms Network

Have you identified a new genetic disease or work on a genetic disease which would merit further research in a model organism? If so, please consult https://rare-diseases-catalyst-network.ca for funding and collaboration opportunities. Letter of Information

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Seeking Farber Disease or SMA-PME Patients

Plexcera Therapeutics LLC, a biotech firm which is developing an enzyme replacement therapy for acid ceramidase deficiency, is seeking Farber Disease patients to be part of a clinical trial to commence tentatively in the second half of 2015.

Dr. Alexander Solyom, MD, working with Plexcera, is collecting natural history data on SMA-PME patients and coordinating an international natural history study of Farber Disease including non-transplanted, transplanted, and patients since deceased.

Please contact him for further information at patients@plexcera.com


Contact Us:
 Please email GarrodAssociation@outlook.com with your questions or comments so they may be directed to the appropriate person.  Thank you.