Evidence map›Paper›PMID 32493524›Full record

Observational studyThe Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques2020

A National Spinal Muscular Atrophy Registry for Real-World Evidence.

Victoria L Hodgkinson, Maryam Oskoui, Joshua Lounsberry, Saïd M'Dahoma, Emily Butler, Craig Campbell, Alex MacKenzie, Hugh J McMillan, Louise Simard, Jiri Vajsar and 40 more

Registry-linked trialAbstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06599606 (Investigation of the Relationship Between Ultrasonographic Muscle Thickness Measurement and Echogenicity Assessment With Functional Scales in Patients With Spinal Muscular Atrophy), which is not on this map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06599606 active not recruitingnot on this mapstarted 2024, after this paper: background citation

Investigation of the Relationship Between Ultrasonographic Muscle Thickness Measurement and Echogenicity Assessment With Functional Scales in Patients With Spinal Muscular Atrophy: An Observational, Cross-Sectional, Case-Control Study

TypeobservationalSponsorIstanbul University - CerrahpasaRan2024 to 2025Enrolled34ConditionsSpinal Muscular Atrophy (SMA), Neuromuscular DiseaseArmsUltrasonographic Assessment
3 · Its place in the literature

Who cites it

11 citing papers in PubMed.

  1. Article
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  4. Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

50 authors.

Victoria L Hodgkinson *Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.ORCID 0000-0001-5908-2694
Maryam Oskoui *Department of Pediatrics, McGill University, Montréal, QC, Canada.
Joshua LounsberryDepartment of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.
Saïd M'DahomaDepartment of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.
Emily ButlerDepartment of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.
Craig CampbellDepartment of Pediatrics, Children's Health Research Institute, University of Western Ontario, London, ON, Canada.
Alex MacKenzieDepartment of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Hugh J McMillanDepartment of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.ORCID 0000-0001-8927-2018
Louise SimardDepartment of Biochemistry and Medical Genetics, University of Manitoba, Winnipeg, MB, Canada.
Jiri VajsarDivision of Neurology, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
Bernard BraisDepartment of Neurology and Neurosurgery and of Pathology, McGill University, Montréal, QC, Canada.
Kristine M ChapmanDivision of Neurology, Department of Medicine, Vancouver General Hospital, University of British Columbia, Vancouver, BC, Canada.
Nicolas ChrestianCHUL Centre Mère-Enfant-Soleil, Laval University, Québec City, QC, Canada.
Meghan CroneDepartment of Pediatrics, Kinsmen Child Centre, University of Saskatchewan, Saskatoon, SK, Canada.
Peter DobrowolskiDivision of Neurology, University of Alberta, Edmonton, AB, Canada.
Susan DojeijiDivision of Physical Medicine and Rehabilitation, Department of Medicine, University of Ottawa, Ottawa, ON, Canada.
James J DowlingDivision of Neurology, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
Nicolas DupréDepartment of Medicine, Laval University, and CHU de Québec-UL, Québec City, QC, Canada.ORCID 0000-0001-7270-6115
Angela GengeDepartment of Neurology and Neurosurgery and of Pathology, McGill University, Montréal, QC, Canada.
Hernan GonorazkyDivision of Neurology, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
Simona HasalDepartment of Pediatrics, Kinsmen Child Centre, University of Saskatchewan, Saskatoon, SK, Canada.
Aaron IzenbergDivision of Neurology, Department of Medicine, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada.
Wendy JohnstonDivision of Neurology, University of Alberta, Edmonton, AB, Canada.
Edward LeungDepartment of Pediatrics and Child Health, University of Manitoba, Winnipeg, MB, Canada.
Hanns LochmüllerDepartment of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Jean K MahDepartment of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.
Alier MarerroCHU Dr. Georges-L-Dumont, and CFNMB, Université de Sherbrooke, Moncton, NB, Canada.
Rami MassieDepartment of Neurology and Neurosurgery and of Pathology, McGill University, Montréal, QC, Canada.ORCID 0000-0001-8219-1020
Laura McAdamDepartment of Pediatrics, Holland Bloorview Kids Rehabilitation Hospital, Bloorview Research Institute, University of Toronto, Toronto, ON, Canada.
Anna McCormickDepartment of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Michel MelansonDepartment of Physical Medicine and Rehabilitation, Queen's University, Kingston, ON, Canada.
Michelle M MezeiDivision of Neurology, Department of Medicine, Vancouver General Hospital, University of British Columbia, Vancouver, BC, Canada.
Cam-Tu E NguyenCHU Sainte-Justine, Université de Montréal, Montréal, QC, Canada.
Colleen O'ConnellStan Cassidy Centre for Rehabilitation, Fredericton, NB, Canada.
Erin K O'FerrallDepartment of Neurology and Neurosurgery and of Pathology, McGill University, Montréal, QC, Canada.
Gerald PfefferDepartment of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.ORCID 0000-0002-7657-7098
Cecile PhanDivision of Neurology, University of Alberta, Edmonton, AB, Canada.
Stephanie PlamondonDepartment of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.
Chantal PoulinDepartment of Pediatrics, McGill University, Montréal, QC, Canada.
Xavier RodrigueDepartment of Medicine, Laval University, and CHU de Québec-UL, Québec City, QC, Canada.
Kerri L SchellenbergDivision of Neurology, Department of Medicine, University of Saskatchewan, Saskatoon, SK, Canada.
Kathy SelbyDivision of Neurology, Department of Pediatrics, BC Children's Hospital, University of Vancouver, Vancouver, BC, Canada.
Jordan SherikoDivision of Neurology, Department of Pediatrics, Dalhousie University, Halifax, NS, Canada.
Christen ShoesmithDivision of Neurology and Clinical Neurological Sciences, London Health Sciences Centre, University of Western Ontario, London, ON, Canada.
Garth SmithDepartment of Pediatrics, KidsInclusive Centre for Child & Youth Development, Hotel Dieu Hospital, Queen's University, Kingston, ON, Canada.
Monique TaillonStan Cassidy Centre for Rehabilitation, Fredericton, NB, Canada.
Sean Taylor
Jodi Warman ChardonDepartment of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Scott WorleyStan Cassidy Centre for Rehabilitation, Fredericton, NB, Canada.
Lawrence KorngutDepartment of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundSpinal muscular atrophy (SMA) is a devastating rare disease that affects individuals regardless of ethnicity, gender, and age. The first-approved disease-modifying therapy for SMA, nusinursen, was approved by Health Canada, as well as by American and European regulatory agencies following positive clinical trial outcomes. The trials were conducted in a narrow pediatric population defined by age, severity, and genotype. Broad approval of therapy necessitates close follow-up of potential rare adverse events and effectiveness in the larger real-world population.

methodsThe Canadian Neuromuscular Disease Registry (CNDR) undertook an iterative multi-stakeholder process to expand the existing SMA dataset to capture items relevant to patient outcomes in a post-marketing environment. The CNDR SMA expanded registry is a longitudinal, prospective, observational study of patients with SMA in Canada designed to evaluate the safety and effectiveness of novel therapies and provide practical information unattainable in trials.

resultsThe consensus expanded dataset includes items that address therapy effectiveness and safety and is collected in a multicenter, prospective, observational study, including SMA patients regardless of therapeutic status. The expanded dataset is aligned with global datasets to facilitate collaboration. Additionally, consensus dataset development aimed to standardize appropriate outcome measures across the network and broader Canadian community. Prospective outcome studies, data use, and analyses are independent of the funding partner.

conclusionProspective outcome data collected will provide results on safety and effectiveness in a post-therapy approval era. These data are essential to inform improvements in care and access to therapy for all SMA patients.

Indexed as

Muscular Atrophy, SpinalCanadaChildHumansProspective StudiesRare DiseasesRegistriesRare diseaseReal-world evidenceRegistrySpinal muscular atrophy

Identifiers

PMID32493524
PMCPMC7656664

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.