The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Througha Nationwide, Pan-NeuromuscularDisease Registry

V. Hodgkinson; J. Lounsberry; S. M'Dahoma; A. Russell; G. Jewett; T. Benstead; B. Brais; C. Campbell; W. Johnston; H. Lochmuller; A. McCormick; C. T. Nguyen; E. O'Ferrall; M. Oskoui; A. Abrahao; H. Briemberg; P. R. Bourque; S. Botez; N. Cashman; K. Chapman; N. Chrestian; M. Crone; P. Dobrowolski; S. Dojeiji; J. J. Dowling; N. Dupre; A. Genge; H. Gonorazky; I. Grant; S. Hasal; A. Izenberg; S. Kalra; H. Katzberg; C. Krieger; E. Leung; G. Linassi; A. Mackenzie; J. K. Mah; A. Marrero; R. Massie; G. Matte; L. McAdam; H. McMillan; M. Melanson; M. M. Mezei; C. O'Connell; G. Pfeffer; C. Phan; S. Plamondon; C. Poulin; X. Rodrigue; K. Schellenberg; K. Selby; J. Sheriko; C. Shoesmith; R. G. Smith; M. Taillon; S. Taylor; S. Venance; J. Warman-Chardon; S. Worley; L. Zinman; L. Korngut

doi:10.3233/JND-200538

The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Througha Nationwide, Pan-NeuromuscularDisease Registry

V. Hodgkinson, J. Lounsberry, S. M'Dahoma, A. Russell, G. Jewett, T. Benstead, B. Brais, C. Campbell, W. Johnston, H. Lochmuller, A. McCormick, C. T. Nguyen, E. O'Ferrall, M. Oskoui, A. Abrahao, H. Briemberg, P. R. Bourque, S. Botez, N. Cashman, K. ChapmanN. Chrestian, M. Crone, P. Dobrowolski, S. Dojeiji, J. J. Dowling, N. Dupre, A. Genge, H. Gonorazky, I. Grant, S. Hasal, A. Izenberg, S. Kalra, H. Katzberg, C. Krieger, E. Leung, G. Linassi, A. Mackenzie, J. K. Mah, A. Marrero, R. Massie, G. Matte, L. McAdam, H. McMillan, M. Melanson, M. M. Mezei, C. O'Connell, G. Pfeffer, C. Phan, S. Plamondon, C. Poulin, X. Rodrigue, K. Schellenberg, K. Selby, J. Sheriko, C. Shoesmith, R. G. Smith, M. Taillon, S. Taylor, S. Venance, J. Warman-Chardon, S. Worley, L. Zinman, L. Korngut

Medicine

Producción científica: Contribución a una revista › Artículo de revisión › revisión exhaustiva

15 Citas (Scopus)

Resumen

We report the recruitment activities and outcomes of a multi-disease neuromuscular patient registry in Canada. The Canadian Neuromuscular Disease Registry (CNDR) registers individuals across Canada with a confirmed diagnosis of a neuromuscular disease. Diagnosis and contact information are collected across all diseases and detailed prospective data is collected for 5 specific diseases: Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy (DM), Limb Girdle Muscular Dystrophy (LGMD), and Spinal Muscular Atrophy (SMA). Since 2010, the CNDR has registered 4306 patients (1154 pediatric and 3148 adult) with 91 different neuromuscular diagnoses and has facilitated 125 projects (73 academic, 3 not-for-profit, 3 government, and 46 commercial) using registry data. In conclusion, the CNDR is an effective and productive pan-neuromuscular registry that has successfully facilitated a substantial number of studies over the past 10 years.

Idioma original	English
Páginas (desde-hasta)	53-61
Número de páginas	9
Publicación	Journal of Neuromuscular Diseases
Volumen	8
N.º	1
DOI	https://doi.org/10.3233/JND-200538
Estado	Published - 2021

Nota bibliográfica

Funding Information:
Dr. Schellenberg received grants for educational activities: Genzyme, Allergan, Mitsubishi-Tanabe; speakers honoraria: Genzyme, EMD Serono, Akcea, and advisory board: Mitsubishi-Tanabe, Alexion, Roche, Biogen, Akcea. Dr. Selby reports grants from Biogen/IONIS, personal fees from Biogen, outside the submitted work. Dr. Sheriko reports personal fees from Biogen, outside the submitted work. Dr. Shoesmith reports other from Biogen, outside the submitted work. Dr. Smith reports personal fees from Shire Pharmaceuticals, personal fees from Purdue Pharmaceuticals, personal fees from Janssen Pharmaceuticals, outside the submitted work. Dr. Taillon reports grants from Allergan, personal fees from Biogen outside the submitted work. Dr. Taylor has nothing to disclose. Dr. Venance has nothing to disclose. Dr. Warman has nothing to disclose. Dr. Worley reports personal fees from Cytokinetics, outside the submitted work. Dr. Zinman reports personal fees from Mitsubishi Tanabe and Biogen outside the submitted work. Dr. Korngut reports grants from Biogen, Sanofi Genzyme, ALS Canada, Jesse’s Journey, during the study, and personal fees from Alexion, Alnylam, Novartis, Mitsubishi Tanabe, Sarepta, Biogen, CSL Behring, outside the submitted work.

Funding Information:
Dr. Hodgkinson reports personal fees from Biogen, Roche, Sarepta, outside the submitted work. Mr. Lounsberry reports personal fees from Biogen Canada Limited, outside the submitted work; Dr. M’Dahoma has nothing to disclose. Ms. Russell has nothing to disclose. Dr. Jewett has nothing to disclose. Dr. Benstead has nothing to disclose. Dr. Campbell reports grants, personal fees and other from Biogen, grants, personal fees and other from PTC Therapeutics, other from Acceleron, AMO, Catabasis, Pfizer, Sarepta, Wave, BMS. Scholar Rock, Roche, outside the submitted work; Dr. McCormick has nothing to disclose. Dr. Johnston declares personal fees from Mitsubishi Tanabe, and clinical trials from Alexion, Cytokinetics, Biogen, Orion, Mallinkrodt, Apellis, Alexion, AB Science; Dr. Nguyen reports other from Pfizer, other from Catabasis outside the submitted work. Dr. O’Ferrall reports personal fees from PTC Therapeutics nmDMD Advisory Board; grants from Sanofi Genzyme, grants from Acceleron, grants from Sanofi Genzyme, grants from Grifols, outside the submitted work. Dr. Oskoui reports grants from Fonds de Recherche Sante du Québec, grants from Kids Brain Health Network, grants from Canadian Institutes of Health Research, grants from SickKids Foundation, grants from Cerebral Palsy Alliance Research Foundation, other from Ionis, other from Biogen, other from Roche, other from Cytokinetics, personal fees and non-financial support from American Academy of Neurology, grants from Fondation du Grand Defi Pierre Lavoie, outside the submitted work; Dr. Brais has nothing to disclose. Dr. Briemberg has nothing to disclose. Dr. Bourque has nothing to disclose. Dr. Botez has nothing to disclose. Dr. Cashman reports ownership of ProMIS Neurosciences, outside the submitted work. Dr. Chapman has nothing to disclose. Dr. Chres-tian received financial support to prepare review courses about SMA for neurologists, Physical therapists and Paediatricians. Dr. Crone reports personal fees from Biogen, outside the submitted work. Dr. Dobrowolski has nothing to disclose. Dr. Dojeiji has nothing to disclose. Dr. Dowling has nothing to disclose. Dr. Dupré has nothing to disclose. Dr. Genge reports grants from Biogen, Sanofi-Genzyme, CSL Behring, MTPA, AL-S Pharma, AB Sciences, Novar-tis, Wave Life Sciences, Argenx, Alexion, Avexis, Ackea, Cytokinetics, Hoffman-La Roche, outside the submitted work. Dr. Gonorazky reports personal fees from Biogen. Dr. Grant has nothing to disclose. Dr. Hasal has nothing to disclose. Dr. Izen-berg has nothing to disclose. Dr. Kalra has nothing to disclose. Dr. Katzberg reports grants from CSL Behring, Takaeda and Muscular Dystrophy Canada and personal fees from Takada, Grifols, CSL Behring, Terumo, Biogen, Pfizer, Akcea, Alexion, Octapharma outside the submitted work. Dr. Krieger has nothing to disclose. Dr. Leung has nothing to disclose. Dr. Linassi has nothing to disclose. Dr. Lochmüller reports grants and personal fees from AMO Pharma, Biogen, Desitin, GW Pharma, Pfizer, PTC Therapeutics, Roche, Santhera, Sarepta, Satellos, Ultragenyx, outside the submitted work;. Dr. MacKenzie reports grants from Biogen, outside the submitted work. Dr. Mah has nothing to disclose. Dr. Marrero has nothing to disclose. Dr. Massie has nothing to disclose. Dr. Matte reports personal fees from Mitsubishi Tanabe, Biogen outside the submitted work, and grants from Biogen, Cytokinetics, Orion, Mallinckrodt outside the submitted work. Dr. McAdam reports other from Italfarmaco, outside the submitted work. Dr. McMil-lan has nothing to disclose. Dr. Melanson has nothing to disclose. Dr. Mezei reports personal fees from Genzyme, Alnylam, Pfizer, Akcea, CSL Behring, outside the submitted work. Dr. O’Connell reports personal fees from MT Pharma, grants and personal fees from Canopy Growth, personal fees from IPSEN, grants from Cytokinetics, grants from Mallincrodtk, grants from Orion, personal fees from Shoppers Drug Mart, outside the submitted work. Dr. Pfeffer has nothing to disclose. Dr. Phan has nothing to disclose. Dr. Plamondon reports other from Biogen, outside the submitted work; Dr. Poulin has nothing to disclose. Dr. Rodrigue has nothing to disclose.

Publisher Copyright:
© 2021 - The authors. Published by IOS Press.

ASJC Scopus Subject Areas

Neurology
Clinical Neurology

PubMed: MeSH publication types

Journal Article

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Hodgkinson, V., Lounsberry, J., M'Dahoma, S., Russell, A., Jewett, G., Benstead, T., Brais, B., Campbell, C., Johnston, W., Lochmuller, H., McCormick, A., Nguyen, C. T., O'Ferrall, E., Oskoui, M., Abrahao, A., Briemberg, H., Bourque, P. R., Botez, S., Cashman, N., ... Korngut, L. (2021). The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Througha Nationwide, Pan-NeuromuscularDisease Registry. Journal of Neuromuscular Diseases, 8(1), 53-61. https://doi.org/10.3233/JND-200538

The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Througha Nationwide, Pan-NeuromuscularDisease Registry. / Hodgkinson, V.; Lounsberry, J.; M'Dahoma, S. et al.
En: Journal of Neuromuscular Diseases, Vol. 8, N.º 1, 2021, p. 53-61.

Producción científica: Contribución a una revista › Artículo de revisión › revisión exhaustiva

Hodgkinson, V, Lounsberry, J, M'Dahoma, S, Russell, A, Jewett, G, Benstead, T, Brais, B, Campbell, C, Johnston, W, Lochmuller, H, McCormick, A, Nguyen, CT, O'Ferrall, E, Oskoui, M, Abrahao, A, Briemberg, H, Bourque, PR, Botez, S, Cashman, N, Chapman, K, Chrestian, N, Crone, M, Dobrowolski, P, Dojeiji, S, Dowling, JJ, Dupre, N, Genge, A, Gonorazky, H, Grant, I, Hasal, S, Izenberg, A, Kalra, S, Katzberg, H, Krieger, C, Leung, E, Linassi, G, Mackenzie, A, Mah, JK, Marrero, A, Massie, R, Matte, G, McAdam, L, McMillan, H, Melanson, M, Mezei, MM, O'Connell, C, Pfeffer, G, Phan, C, Plamondon, S, Poulin, C, Rodrigue, X, Schellenberg, K, Selby, K, Sheriko, J, Shoesmith, C, Smith, RG, Taillon, M, Taylor, S, Venance, S, Warman-Chardon, J, Worley, S, Zinman, L & Korngut, L 2021, 'The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Througha Nationwide, Pan-NeuromuscularDisease Registry', Journal of Neuromuscular Diseases, vol. 8, n.º 1, pp. 53-61. https://doi.org/10.3233/JND-200538

@article{ca17370f44b3488abb49fa37556a0141,

title = "The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Througha Nationwide, Pan-NeuromuscularDisease Registry",

abstract = "We report the recruitment activities and outcomes of a multi-disease neuromuscular patient registry in Canada. The Canadian Neuromuscular Disease Registry (CNDR) registers individuals across Canada with a confirmed diagnosis of a neuromuscular disease. Diagnosis and contact information are collected across all diseases and detailed prospective data is collected for 5 specific diseases: Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy (DM), Limb Girdle Muscular Dystrophy (LGMD), and Spinal Muscular Atrophy (SMA). Since 2010, the CNDR has registered 4306 patients (1154 pediatric and 3148 adult) with 91 different neuromuscular diagnoses and has facilitated 125 projects (73 academic, 3 not-for-profit, 3 government, and 46 commercial) using registry data. In conclusion, the CNDR is an effective and productive pan-neuromuscular registry that has successfully facilitated a substantial number of studies over the past 10 years.",

author = "V. Hodgkinson and J. Lounsberry and S. M'Dahoma and A. Russell and G. Jewett and T. Benstead and B. Brais and C. Campbell and W. Johnston and H. Lochmuller and A. McCormick and Nguyen, {C. T.} and E. O'Ferrall and M. Oskoui and A. Abrahao and H. Briemberg and Bourque, {P. R.} and S. Botez and N. Cashman and K. Chapman and N. Chrestian and M. Crone and P. Dobrowolski and S. Dojeiji and Dowling, {J. J.} and N. Dupre and A. Genge and H. Gonorazky and I. Grant and S. Hasal and A. Izenberg and S. Kalra and H. Katzberg and C. Krieger and E. Leung and G. Linassi and A. Mackenzie and Mah, {J. K.} and A. Marrero and R. Massie and G. Matte and L. McAdam and H. McMillan and M. Melanson and Mezei, {M. M.} and C. O'Connell and G. Pfeffer and C. Phan and S. Plamondon and C. Poulin and X. Rodrigue and K. Schellenberg and K. Selby and J. Sheriko and C. Shoesmith and Smith, {R. G.} and M. Taillon and S. Taylor and S. Venance and J. Warman-Chardon and S. Worley and L. Zinman and L. Korngut",

note = "Funding Information: Dr. Schellenberg received grants for educational activities: Genzyme, Allergan, Mitsubishi-Tanabe; speakers honoraria: Genzyme, EMD Serono, Akcea, and advisory board: Mitsubishi-Tanabe, Alexion, Roche, Biogen, Akcea. Dr. Selby reports grants from Biogen/IONIS, personal fees from Biogen, outside the submitted work. Dr. Sheriko reports personal fees from Biogen, outside the submitted work. Dr. Shoesmith reports other from Biogen, outside the submitted work. Dr. Smith reports personal fees from Shire Pharmaceuticals, personal fees from Purdue Pharmaceuticals, personal fees from Janssen Pharmaceuticals, outside the submitted work. Dr. Taillon reports grants from Allergan, personal fees from Biogen outside the submitted work. Dr. Taylor has nothing to disclose. Dr. Venance has nothing to disclose. Dr. Warman has nothing to disclose. Dr. Worley reports personal fees from Cytokinetics, outside the submitted work. Dr. Zinman reports personal fees from Mitsubishi Tanabe and Biogen outside the submitted work. Dr. Korngut reports grants from Biogen, Sanofi Genzyme, ALS Canada, Jesse{\textquoteright}s Journey, during the study, and personal fees from Alexion, Alnylam, Novartis, Mitsubishi Tanabe, Sarepta, Biogen, CSL Behring, outside the submitted work. Funding Information: Dr. Hodgkinson reports personal fees from Biogen, Roche, Sarepta, outside the submitted work. Mr. Lounsberry reports personal fees from Biogen Canada Limited, outside the submitted work; Dr. M{\textquoteright}Dahoma has nothing to disclose. Ms. Russell has nothing to disclose. Dr. Jewett has nothing to disclose. Dr. Benstead has nothing to disclose. Dr. Campbell reports grants, personal fees and other from Biogen, grants, personal fees and other from PTC Therapeutics, other from Acceleron, AMO, Catabasis, Pfizer, Sarepta, Wave, BMS. Scholar Rock, Roche, outside the submitted work; Dr. McCormick has nothing to disclose. Dr. Johnston declares personal fees from Mitsubishi Tanabe, and clinical trials from Alexion, Cytokinetics, Biogen, Orion, Mallinkrodt, Apellis, Alexion, AB Science; Dr. Nguyen reports other from Pfizer, other from Catabasis outside the submitted work. Dr. O{\textquoteright}Ferrall reports personal fees from PTC Therapeutics nmDMD Advisory Board; grants from Sanofi Genzyme, grants from Acceleron, grants from Sanofi Genzyme, grants from Grifols, outside the submitted work. Dr. Oskoui reports grants from Fonds de Recherche Sante du Qu{\'e}bec, grants from Kids Brain Health Network, grants from Canadian Institutes of Health Research, grants from SickKids Foundation, grants from Cerebral Palsy Alliance Research Foundation, other from Ionis, other from Biogen, other from Roche, other from Cytokinetics, personal fees and non-financial support from American Academy of Neurology, grants from Fondation du Grand Defi Pierre Lavoie, outside the submitted work; Dr. Brais has nothing to disclose. Dr. Briemberg has nothing to disclose. Dr. Bourque has nothing to disclose. Dr. Botez has nothing to disclose. Dr. Cashman reports ownership of ProMIS Neurosciences, outside the submitted work. Dr. Chapman has nothing to disclose. Dr. Chres-tian received financial support to prepare review courses about SMA for neurologists, Physical therapists and Paediatricians. Dr. Crone reports personal fees from Biogen, outside the submitted work. Dr. Dobrowolski has nothing to disclose. Dr. Dojeiji has nothing to disclose. Dr. Dowling has nothing to disclose. Dr. Dupr{\'e} has nothing to disclose. Dr. Genge reports grants from Biogen, Sanofi-Genzyme, CSL Behring, MTPA, AL-S Pharma, AB Sciences, Novar-tis, Wave Life Sciences, Argenx, Alexion, Avexis, Ackea, Cytokinetics, Hoffman-La Roche, outside the submitted work. Dr. Gonorazky reports personal fees from Biogen. Dr. Grant has nothing to disclose. Dr. Hasal has nothing to disclose. Dr. Izen-berg has nothing to disclose. Dr. Kalra has nothing to disclose. Dr. Katzberg reports grants from CSL Behring, Takaeda and Muscular Dystrophy Canada and personal fees from Takada, Grifols, CSL Behring, Terumo, Biogen, Pfizer, Akcea, Alexion, Octapharma outside the submitted work. Dr. Krieger has nothing to disclose. Dr. Leung has nothing to disclose. Dr. Linassi has nothing to disclose. Dr. Lochm{\"u}ller reports grants and personal fees from AMO Pharma, Biogen, Desitin, GW Pharma, Pfizer, PTC Therapeutics, Roche, Santhera, Sarepta, Satellos, Ultragenyx, outside the submitted work;. Dr. MacKenzie reports grants from Biogen, outside the submitted work. Dr. Mah has nothing to disclose. Dr. Marrero has nothing to disclose. Dr. Massie has nothing to disclose. Dr. Matte reports personal fees from Mitsubishi Tanabe, Biogen outside the submitted work, and grants from Biogen, Cytokinetics, Orion, Mallinckrodt outside the submitted work. Dr. McAdam reports other from Italfarmaco, outside the submitted work. Dr. McMil-lan has nothing to disclose. Dr. Melanson has nothing to disclose. Dr. Mezei reports personal fees from Genzyme, Alnylam, Pfizer, Akcea, CSL Behring, outside the submitted work. Dr. O{\textquoteright}Connell reports personal fees from MT Pharma, grants and personal fees from Canopy Growth, personal fees from IPSEN, grants from Cytokinetics, grants from Mallincrodtk, grants from Orion, personal fees from Shoppers Drug Mart, outside the submitted work. Dr. Pfeffer has nothing to disclose. Dr. Phan has nothing to disclose. Dr. Plamondon reports other from Biogen, outside the submitted work; Dr. Poulin has nothing to disclose. Dr. Rodrigue has nothing to disclose. Publisher Copyright: {\textcopyright} 2021 - The authors. Published by IOS Press.",

year = "2021",

doi = "10.3233/JND-200538",

language = "English",

volume = "8",

pages = "53--61",

journal = "Journal of Neuromuscular Diseases",

issn = "2214-3599",

publisher = "IOS Press BV",

number = "1",

}

TY - JOUR

T1 - The Canadian Neuromuscular Disease Registry 2010-2019

T2 - A Decade of Facilitating Clinical Research Througha Nationwide, Pan-NeuromuscularDisease Registry

AU - Hodgkinson, V.

AU - Lounsberry, J.

AU - M'Dahoma, S.

AU - Russell, A.

AU - Jewett, G.

AU - Benstead, T.

AU - Brais, B.

AU - Campbell, C.

AU - Johnston, W.

AU - Lochmuller, H.

AU - McCormick, A.

AU - Nguyen, C. T.

AU - O'Ferrall, E.

AU - Oskoui, M.

AU - Abrahao, A.

AU - Briemberg, H.

AU - Bourque, P. R.

AU - Botez, S.

AU - Cashman, N.

AU - Chapman, K.

AU - Chrestian, N.

AU - Crone, M.

AU - Dobrowolski, P.

AU - Dojeiji, S.

AU - Dowling, J. J.

AU - Dupre, N.

AU - Genge, A.

AU - Gonorazky, H.

AU - Grant, I.

AU - Hasal, S.

AU - Izenberg, A.

AU - Kalra, S.

AU - Katzberg, H.

AU - Krieger, C.

AU - Leung, E.

AU - Linassi, G.

AU - Mackenzie, A.

AU - Mah, J. K.

AU - Marrero, A.

AU - Massie, R.

AU - Matte, G.

AU - McAdam, L.

AU - McMillan, H.

AU - Melanson, M.

AU - Mezei, M. M.

AU - O'Connell, C.

AU - Pfeffer, G.

AU - Phan, C.

AU - Plamondon, S.

AU - Poulin, C.

AU - Rodrigue, X.

AU - Schellenberg, K.

AU - Selby, K.

AU - Sheriko, J.

AU - Shoesmith, C.

AU - Smith, R. G.

AU - Taillon, M.

AU - Taylor, S.

AU - Venance, S.

AU - Warman-Chardon, J.

AU - Worley, S.

AU - Zinman, L.

AU - Korngut, L.

N1 - Funding Information: Dr. Schellenberg received grants for educational activities: Genzyme, Allergan, Mitsubishi-Tanabe; speakers honoraria: Genzyme, EMD Serono, Akcea, and advisory board: Mitsubishi-Tanabe, Alexion, Roche, Biogen, Akcea. Dr. Selby reports grants from Biogen/IONIS, personal fees from Biogen, outside the submitted work. Dr. Sheriko reports personal fees from Biogen, outside the submitted work. Dr. Shoesmith reports other from Biogen, outside the submitted work. Dr. Smith reports personal fees from Shire Pharmaceuticals, personal fees from Purdue Pharmaceuticals, personal fees from Janssen Pharmaceuticals, outside the submitted work. Dr. Taillon reports grants from Allergan, personal fees from Biogen outside the submitted work. Dr. Taylor has nothing to disclose. Dr. Venance has nothing to disclose. Dr. Warman has nothing to disclose. Dr. Worley reports personal fees from Cytokinetics, outside the submitted work. Dr. Zinman reports personal fees from Mitsubishi Tanabe and Biogen outside the submitted work. Dr. Korngut reports grants from Biogen, Sanofi Genzyme, ALS Canada, Jesse’s Journey, during the study, and personal fees from Alexion, Alnylam, Novartis, Mitsubishi Tanabe, Sarepta, Biogen, CSL Behring, outside the submitted work. Funding Information: Dr. Hodgkinson reports personal fees from Biogen, Roche, Sarepta, outside the submitted work. Mr. Lounsberry reports personal fees from Biogen Canada Limited, outside the submitted work; Dr. M’Dahoma has nothing to disclose. Ms. Russell has nothing to disclose. Dr. Jewett has nothing to disclose. Dr. Benstead has nothing to disclose. Dr. Campbell reports grants, personal fees and other from Biogen, grants, personal fees and other from PTC Therapeutics, other from Acceleron, AMO, Catabasis, Pfizer, Sarepta, Wave, BMS. Scholar Rock, Roche, outside the submitted work; Dr. McCormick has nothing to disclose. Dr. Johnston declares personal fees from Mitsubishi Tanabe, and clinical trials from Alexion, Cytokinetics, Biogen, Orion, Mallinkrodt, Apellis, Alexion, AB Science; Dr. Nguyen reports other from Pfizer, other from Catabasis outside the submitted work. Dr. O’Ferrall reports personal fees from PTC Therapeutics nmDMD Advisory Board; grants from Sanofi Genzyme, grants from Acceleron, grants from Sanofi Genzyme, grants from Grifols, outside the submitted work. Dr. Oskoui reports grants from Fonds de Recherche Sante du Québec, grants from Kids Brain Health Network, grants from Canadian Institutes of Health Research, grants from SickKids Foundation, grants from Cerebral Palsy Alliance Research Foundation, other from Ionis, other from Biogen, other from Roche, other from Cytokinetics, personal fees and non-financial support from American Academy of Neurology, grants from Fondation du Grand Defi Pierre Lavoie, outside the submitted work; Dr. Brais has nothing to disclose. Dr. Briemberg has nothing to disclose. Dr. Bourque has nothing to disclose. Dr. Botez has nothing to disclose. Dr. Cashman reports ownership of ProMIS Neurosciences, outside the submitted work. Dr. Chapman has nothing to disclose. Dr. Chres-tian received financial support to prepare review courses about SMA for neurologists, Physical therapists and Paediatricians. Dr. Crone reports personal fees from Biogen, outside the submitted work. Dr. Dobrowolski has nothing to disclose. Dr. Dojeiji has nothing to disclose. Dr. Dowling has nothing to disclose. Dr. Dupré has nothing to disclose. Dr. Genge reports grants from Biogen, Sanofi-Genzyme, CSL Behring, MTPA, AL-S Pharma, AB Sciences, Novar-tis, Wave Life Sciences, Argenx, Alexion, Avexis, Ackea, Cytokinetics, Hoffman-La Roche, outside the submitted work. Dr. Gonorazky reports personal fees from Biogen. Dr. Grant has nothing to disclose. Dr. Hasal has nothing to disclose. Dr. Izen-berg has nothing to disclose. Dr. Kalra has nothing to disclose. Dr. Katzberg reports grants from CSL Behring, Takaeda and Muscular Dystrophy Canada and personal fees from Takada, Grifols, CSL Behring, Terumo, Biogen, Pfizer, Akcea, Alexion, Octapharma outside the submitted work. Dr. Krieger has nothing to disclose. Dr. Leung has nothing to disclose. Dr. Linassi has nothing to disclose. Dr. Lochmüller reports grants and personal fees from AMO Pharma, Biogen, Desitin, GW Pharma, Pfizer, PTC Therapeutics, Roche, Santhera, Sarepta, Satellos, Ultragenyx, outside the submitted work;. Dr. MacKenzie reports grants from Biogen, outside the submitted work. Dr. Mah has nothing to disclose. Dr. Marrero has nothing to disclose. Dr. Massie has nothing to disclose. Dr. Matte reports personal fees from Mitsubishi Tanabe, Biogen outside the submitted work, and grants from Biogen, Cytokinetics, Orion, Mallinckrodt outside the submitted work. Dr. McAdam reports other from Italfarmaco, outside the submitted work. Dr. McMil-lan has nothing to disclose. Dr. Melanson has nothing to disclose. Dr. Mezei reports personal fees from Genzyme, Alnylam, Pfizer, Akcea, CSL Behring, outside the submitted work. Dr. O’Connell reports personal fees from MT Pharma, grants and personal fees from Canopy Growth, personal fees from IPSEN, grants from Cytokinetics, grants from Mallincrodtk, grants from Orion, personal fees from Shoppers Drug Mart, outside the submitted work. Dr. Pfeffer has nothing to disclose. Dr. Phan has nothing to disclose. Dr. Plamondon reports other from Biogen, outside the submitted work; Dr. Poulin has nothing to disclose. Dr. Rodrigue has nothing to disclose. Publisher Copyright: © 2021 - The authors. Published by IOS Press.

PY - 2021

Y1 - 2021

N2 - We report the recruitment activities and outcomes of a multi-disease neuromuscular patient registry in Canada. The Canadian Neuromuscular Disease Registry (CNDR) registers individuals across Canada with a confirmed diagnosis of a neuromuscular disease. Diagnosis and contact information are collected across all diseases and detailed prospective data is collected for 5 specific diseases: Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy (DM), Limb Girdle Muscular Dystrophy (LGMD), and Spinal Muscular Atrophy (SMA). Since 2010, the CNDR has registered 4306 patients (1154 pediatric and 3148 adult) with 91 different neuromuscular diagnoses and has facilitated 125 projects (73 academic, 3 not-for-profit, 3 government, and 46 commercial) using registry data. In conclusion, the CNDR is an effective and productive pan-neuromuscular registry that has successfully facilitated a substantial number of studies over the past 10 years.

AB - We report the recruitment activities and outcomes of a multi-disease neuromuscular patient registry in Canada. The Canadian Neuromuscular Disease Registry (CNDR) registers individuals across Canada with a confirmed diagnosis of a neuromuscular disease. Diagnosis and contact information are collected across all diseases and detailed prospective data is collected for 5 specific diseases: Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy (DM), Limb Girdle Muscular Dystrophy (LGMD), and Spinal Muscular Atrophy (SMA). Since 2010, the CNDR has registered 4306 patients (1154 pediatric and 3148 adult) with 91 different neuromuscular diagnoses and has facilitated 125 projects (73 academic, 3 not-for-profit, 3 government, and 46 commercial) using registry data. In conclusion, the CNDR is an effective and productive pan-neuromuscular registry that has successfully facilitated a substantial number of studies over the past 10 years.

UR - http://www.scopus.com/inward/record.url?scp=85098714037&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85098714037&partnerID=8YFLogxK

U2 - 10.3233/JND-200538

DO - 10.3233/JND-200538

M3 - Review article

C2 - 32925088

AN - SCOPUS:85098714037

SN - 2214-3599

VL - 8

SP - 53

EP - 61

JO - Journal of Neuromuscular Diseases

JF - Journal of Neuromuscular Diseases

IS - 1

ER -

The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Througha Nationwide, Pan-NeuromuscularDisease Registry

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