Evidence map›Paper›PMID 40770363›Full record

ArticleOrphanet journal of rare diseases2025

Assessing the socio-economic burden of inherited and inflammatory neuromuscular diseases (BIND study): a study protocol.

Ian C Smith, Yasmin Abusetah, Homira Osman, Aditi Garg, Alyssa Grant, Hanns Lochmuller, Hugh McMillan, Gerald Pfeffer, Lawrence Korngut, Cynthia Gagnon and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Article
  3. Counting the Cost: The Hidden Financial Realities of Neuromuscular Disease Through Patient and Family Perspectives.Health expectations : an international journal of public participation in health care and health policy · 2025
    Article
  4. Article
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

15 authors.

Ian C Smith *The Ottawa Hospital Research Institute, Ottawa, ON, Canada.ORCID http://orcid.org/0000-0001-5269-1710
Yasmin Abusetah *Faculty of Science, University of Ottawa, Ottawa, ON, Canada.ORCID http://orcid.org/0009-0001-7294-5105
Homira OsmanMuscular Dystrophy Canada, Toronto, ON, Canada.ORCID http://orcid.org/0009-0001-4225-9075
Aditi GargUniversity of Ottawa Heart Institute, Ottawa, ON, Canada.ORCID http://orcid.org/0009-0005-7095-2407
Alyssa GrantThe Ottawa Hospital Research Institute, Ottawa, ON, Canada.ORCID http://orcid.org/0000-0003-3374-5212
Hanns LochmullerThe Ottawa Hospital Research Institute, Ottawa, ON, Canada.ORCID http://orcid.org/0000-0003-2324-8001
Hugh McMillanFaculty of Medicine, University of Ottawa, Ottawa, ON, Canada.ORCID http://orcid.org/0000-0001-8927-2018
Gerald PfefferDepartment of Clinical Neurosciences, Cumming School of Medicine, Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.ORCID http://orcid.org/0000-0002-7657-7098
Lawrence KorngutDepartment of Clinical Neurosciences, Cumming School of Medicine, Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada.ORCID http://orcid.org/0000-0003-1594-5923
Cynthia GagnonUniversity of Sherbrooke, Sherbrooke, QC, Canada.ORCID http://orcid.org/0000-0003-2829-8470
Stacey LinternMuscular Dystrophy Canada, Toronto, ON, Canada.
Daria WojtalMuscular Dystrophy Canada, Toronto, ON, Canada.ORCID http://orcid.org/0000-0002-3401-4641
Kathy SelbyBritish Columbia Children's Hospital, Vancouver, BC, Canada.ORCID http://orcid.org/0000-0001-9239-1704
Kednapa ThavornThe Ottawa Hospital Research Institute, Ottawa, ON, Canada. kthavorn@ohri.ca.ORCID http://orcid.org/0000-0003-4738-8447
Jodi Warman-ChardonThe Ottawa Hospital Research Institute, Ottawa, ON, Canada.ORCID http://orcid.org/0000-0002-0187-2199

Funding

Canada First Research Excellence Fund CFREF-2022-00007Canada Foundation for Innovation CFI-JELF 38412Canada Research Chairs 950-232279Canada Research Coordinating Committee NFRFG-2022-00033CIHR 178534CIHR ERT-174211CIHR FDN-167281CIHR INS-464765CIHR OR2-189333European Commission 101080249Muscular Dystrophy Canada 932196Physicians' Services Incorporated Foundation 19-28
6 · The paper itself

Abstract

introductionNeuromuscular diseases (NMDs) are rare multisystem, genetic or acquired disorders causing weakness and/or sensory loss. It is essential for governments, insurance providers, and broader society to have a better understanding of the burden of illness of NMDs. Our goal is to assess the social and economic burden of Canadians living with NMDs, encompassing schooling and education achievement, health-related quality-of-life, and labour force participation and productivity. METHODS AND ANALYSIS: We will conduct a national, cross-sectional survey of individuals living with a NMD and their caregivers who are members of Muscular Dystrophy Canada and/or are patients within our national network of neuromuscular clinics. Surveys can be completed online or via telephone. The specific sub-sections of the questionnaire will differ based on respondent's profile, whether they are 1) a minor living with a NMD, 2) an adult living with a NMD, 3) an adult who is a caregiver for someone living with a NMD, or 4) an adult who both lives with a NMD and is a caregiver for someone with a NMD. We will use descriptive statistics to describe distributions and ranges of the social and economic measures. Pearson correlations for continuous data and Spearman rho for rank data will be used to detect the strength of association of socio-demographic factors, disease characteristics, and social and economic impacts of NMDs. ETHICS AND DISSEMINATION: The study protocol has been approved by the Ottawa Health Science Network Research Ethics Board (Protocol ID # 20210601-01H). This study will provide the overall impact of NMD on costs and health-related quality of life, disseminated via a series of manuscripts which will include both between- and within-NMD/NMD subtype comparisons. The data obtained will guide governmental policy development and inform patient organisation programs to deliver more effective supports to individuals and families affected by NMDs.

Indexed as

InflammationNeuromuscular DiseasesAdultCanadaCaregiversCost of IllnessCross-Sectional StudiesFemaleHumansQuality of LifeSurveys and QuestionnairesCross-sectional surveyNeuromuscular diseaseSocio-economic burden

Identifiers

PMID40770363
PMCPMC12326678

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.