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The CNDR: Collaborating to Translate New Therapies for Canadians

Published online by Cambridge University Press:  23 September 2014

Lawrence Korngut*
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Craig Campbell
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Megan Johnston
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Timothy Benstead
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Angela Genge
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Alex MacKenzie
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Anna McCormick
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Douglas Biggar
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Pierre Bourque
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Hannah Briemberg
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Colleen O'Connell
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Suzan Dojeiji
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Joseph Dooley
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Ian Grant
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Gillian Hogan
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Wendy Johnston
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Sanjay Kalra
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Hans D. Katzberg
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Jean K. Mah
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Laura McAdam
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Hugh J. McMillan
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Michel Melanson
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Kathryn Selby
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Christen Shoesmith
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Garth Smith
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Shannon L. Venance
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
Joy Wee
Affiliation:
Canadian Neuromuscular Disease Registry Investigator Network
*
Clinical Neurosciences, 480060, 4th Floor Administration, South Health Campus, 4448 Front Street SE, Calgary, Alberta, T3M 1M4, Canada. Email: lawrence.korngut@albertahealthservices.ca
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Abstract:

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Background:

Patient registries represent an important method of organizing “real world” patient information for clinical and research purposes. Registries can facilitate clinical trial planning and recruitment and are particularly useful in this regard for uncommon and rare diseases. Neuromuscular diseases (NMDs) are individually rare but in aggregate have a significant prevalence. In Canada, information on NMDs is lacking. Barriers to performing Canadian multicentre NMD research exist which can be overcome by a comprehensive and collaborative NMD registry.

Methods:

We describe the objectives, design, feasibility and initial recruitment results for the Canadian Neuromuscular Disease Registry (CNDR).

Results:

The CNDR is a clinic-based registry which launched nationally in June 2011, incorporates paediatric and adult neuromuscular clinics in British Columbia, Alberta, Ontario, Quebec, New Brunswick and Nova Scotia and, as of December 2012, has recruited 1161 patients from 12 provinces and territories. Complete medical datasets have been captured on 460 “index disease” patients. Another 618 “non-index” patients have been recruited with capture of physician-confirmed diagnosis and contact information. We have demonstrated the feasibility of blended clinic and central office-based recruitment. “Index disease” patients recruited at the time of writing include 253 with Duchenne and Becker muscular dystrophy, 161 with myotonic dystrophy, and 71 with ALS.

Conclusions:

The CNDR is a new nationwide registry of patients with NMDs that represents an important advance in Canadian neuromuscular disease research capacity. It provides an innovative platform for organizing patient information to facilitate clinical research and to expedite translation of recent laboratory findings into human studies.

Type
Original Article
Copyright
Copyright © The Canadian Journal of Neurological 2013

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