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The CNDR: Collaborating to Translate New Therapies for Canadians

Published online by Cambridge University Press:  23 September 2014

Lawrence Korngut ,
Craig Campbell ,
Megan Johnston ,
Timothy Benstead ,
Angela Genge ,
Alex MacKenzie ,
Anna McCormick ,
Douglas Biggar ,
Pierre Bourque  and
Hannah Briemberg
...Show all authors
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: [email protected]
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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
Information
Canadian Journal of Neurological Sciences , Volume 40 , Issue 5 , September 2013 , pp. 698 - 704
Copyright
Copyright © The Canadian Journal of Neurological 2013

References

1.Shaw, JE, Sicree, RA, Zimmet, PZ.Global estimates of the prevalence of diabetes for 2010 and 2030. Diabetes Res Clin Pract. 2010;87(1):4–14.Google Scholar
2.Karvestedt, L, Martensson, E, Grill, V, et al.The prevalence of peripheral neuropathy in a population-based study of patients with type 2 diabetes in Sweden. J Diabetes Complications. 2011;25(2):97–106.CrossRefGoogle Scholar
3.Hughes, MI, Hicks, EM, Nevin, NC, Patterson, VH.The prevalence of inherited neuromuscular disease in Northern Ireland. Neuromuscular disorders: NMD. 1996;6(1):69–73.Google Scholar
4.Johnston, CA, Stanton, BR, Turner, MR, et al.Amyotrophic lateral sclerosis in an urban setting: a population based study of inner city London. J Neurol. 2006;253(12):1642–3.Google Scholar
5.Bushby, K, Lynn, S, Straub, T, TREAT-NMD Network. Collaborating to bring new therapies to the patient-the TREAT-NMD model. Acta Myol. 2009;(1):12–15.Google Scholar
6.Mah, JK, Selby, K, Campbell, C, et al.A population-based study of dystrophin mutations in Canada. Can J Neurol Sci. 2011 May;38(3):465–74.CrossRefGoogle ScholarPubMed
7.Aggarwal, SP, Zinman, L, Simpson, E, et al.Safety and efficacy of lithium in combination with riluzole for treatment of amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2010;9:481–8.Google Scholar
8.Donaghy, C, Clarke, J, Patterson, C, Kee, F, Hardiman, O, Patterson, V.The epidemiology of motor neuron disease in Northern Ireland using capture-recapture methodology. Amyotroph Lateral Scler. 2010;(4):374–8.Google Scholar
9.Lijovic, M, Davis, SR, Fradkin, P, et al.Use of a cancer registry is preferable to a direct-to-community approach for recruitment to a cohort study of wellbeing in women newly diagnosed with invasive breast cancer. BMC Cancer. 2008;8:126.Google Scholar
10.Lemmers, RJLF, van der Vliet, PJ, Klooster, R, et al.A unifying genetic model for facioscapulohumeral muscular dystrophy. Science. 2010;329(5999):1650–3.Google Scholar
11.Deng, H-X, Chen, W, Hong, S-T, et al.Mutations in UBQLN2 cause dominant X-linked juvenile and adult-onset ALS and ALS/dementia. Nature. 2011;477(7363):211–15.CrossRefGoogle ScholarPubMed
12.Strong, MJ.The evidence for altered RNA metabolism in amyotrophic lateral sclerosis (ALS). J Neurol Sci. 2010;288(1-2):1–12.Google Scholar
13.López Castel, A, Cleary, JD, Pearson, CE.Repeat instability as the basis for human diseases and as a potential target for therapy. Nat Rev Mol Cell Biol. 2010 March;11(3):165–70.Google Scholar
14.Kinali, M, Arechavala-Gomeza, V, Feng, L, et al.Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol. 2009;8(10):918–28.CrossRefGoogle ScholarPubMed
15.Goemans, NM, Tulinius, M, van den Akker, JT, et al.Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med. 2011 April 4;364(16):1513–22.CrossRefGoogle ScholarPubMed
16.Kishnani, PS, Corzo, D, Leslie, ND, et al.Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease. Pediatr Res. 2009 September;66(3):329–35.Google Scholar
17.Rowland, LP, Mcleod, JG.Classification of neuromuscular disorders. J Neurol Sci. 1994;124:109.Google ScholarPubMed
18.Thompson, R, Schoser, B, Monckton, DG, Blonsky, K, Lochmüller, H.Patient Registries and Trial Readiness in Myotonic Dystrophy-TREAT-NMD/Marigold International Workshop Report. Neuromuscular disorders: NMD. 2009;19(12):860.Google Scholar
19.Centers for Disease Disease, Control, Prevention. Prevalence of Duchenne/Becker muscular dystrophy among males aged 5-24 years - four states, 2007. MMWR Morb Mortal Wkly Rep. 2009;58(40):1119–22.Google Scholar