The CNDR: Collaborating to translate new therapies for Canadians

Authors

Lawrence Korngut, Canadian Neuromuscular Disease Registry Investigator Network
Craig Campbell, Canadian Neuromuscular Disease Registry Investigator NetworkFollow
Megan Johnston, Canadian Neuromuscular Disease Registry Investigator Network
Timothy Benstead, Canadian Neuromuscular Disease Registry Investigator Network
Angela Genge, Canadian Neuromuscular Disease Registry Investigator Network
Alex MacKenzie, Canadian Neuromuscular Disease Registry Investigator Network
Anna McCormick, Canadian Neuromuscular Disease Registry Investigator Network
Douglas Biggar, Canadian Neuromuscular Disease Registry Investigator Network

Document Type

Article

Publication Date

9-1-2013

Journal

Canadian Journal of Neurological Sciences

Volume

40

Issue

5

First Page

698

Last Page

704

URL with Digital Object Identifier

10.1017/S0317167100014943

Abstract

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.