{ "abstract": "Introduction: Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD) are progressive genetic neuromuscular disorders that impose substantial caregiving demands on families. In Pakistan, where disease-modifying therapies remain largely inaccessible, care is predominantly supportive, frequently resulting in psychological distress and diminished caregiver confidence. An individual's belief in their capacity to execute required behaviours, self-efficacy, is a recognised determinant of caregiving quality, yet evidence on caregiver self-efficacy in SMA and DMD within low-resource settings remains sparse. This protocol describes the methods for an exploratory, single-arm, pre-post trial. The trial aims to generate preliminary evidence on changes in self-efficacy following a multicomponent, digitally delivered intervention and its implementation feasibility among primary caregivers of children with SMA and DMD in Pakistan. Methods and analysis: A single-arm, pre-post study design will recruit primary caregivers of children with SMA and DMD enrolled in the Treat-NMD Registry of Pakistan, with 40 caregivers per condition (80 total) analyzed as separate disease-specific cohorts. An eight-week intervention will be delivered via online support groups, comprising educational video clips, expert-led live sessions, pictorial guides, and progressively tapering audio reminders. The primary outcome, self-efficacy, will be measured using a culturally adapted, content-validated Urdu version of the DMD Caregiver Self-Efficacy Scale (DMD-CSES), assessed at baseline (T0) and eight weeks post-intervention (T1). Pre- and post-intervention scores will be compared using a paired t-test or Wilcoxon signed-rank test, depending on the data distribution, with analyses conducted in STATA Version 17. Ethics and dissemination: Ethical approval has been granted by the Ethics Review Committee of Aga Khan University (2025-11875-37040). Verbal informed consent will be obtained from all participants, and confidentiality will be maintained throughout the trial. The findings will be disseminated via peer-reviewed publications, conference presentations, and shared with the Treat-NMD registry network. Trial registration name and number: NCT07356063, Date of registration: 11/January/2026.", "abstract_shown": true, "affiliations": [ "Brain and Mind Institute, Aga Khan University, Karachi, Sindh, Pakistan", "Department of Community Health Sciences, Aga Khan University, Karachi, Sindh, Pakistan", "Department of Medicine, Section of Neurology, Aga Khan University, Karachi, Sindh, Pakistan" ], "article_type": "Study Protocol", "arxiv": null, "author_total": 3, "authors": [ { "affs": [ 0 ], "name": "Nashit Irfan Aziz" }, { "affs": [ 1 ], "name": "Wardah Khalid" }, { "affs": [ 2 ], "name": "Sara Khan" } ], "bibtex": "https://research.draconitic.com/paper/6bcjd2xs6hm07.bib", "cited_by": 0, "doi": "10.1371/journal.pone.0349543", "fulltext_url": null, "id": "6bcjd2xs6hm07", "issns": [ "1932-6203" ], "landing_url": "https://doi.org/10.1371/journal.pone.0349543", "language": null, "licence": "CC-BY-4.0", "licence_label": "CC BY 4.0", "licence_source": "journal", "licence_url": "https://creativecommons.org/licenses/by/4.0/", "metadata_licence": null, "native_subjects": [ [ "openalex", "2716|Neurogenetic and Muscular Disorders Research" ], [ "plos", "/Biology and life sciences/Genetics/Heredity/Genetic linkage/Sex linkage/X-linked traits/Duchenne muscular dystrophy" ], [ "plos", "/Biology and life sciences/Neuroscience/Cognitive science/Cognitive psychology/Learning/Human learning" ], [ "plos", "/Biology and life sciences/Neuroscience/Learning and memory/Learning/Human learning" ], [ "plos", "/Biology and life sciences/Psychology/Cognitive psychology/Learning/Human learning" ], [ "plos", "/Biology and life sciences/Psychology/Psychometrics" ], [ "plos", "/Medicine and health sciences/Clinical genetics/X-linked traits/Duchenne muscular dystrophy" ], [ "plos", "/Medicine and health sciences/Epidemiology/Medical risk factors" ], [ "plos", "/Medicine and health sciences/Health care/Caregivers" ], [ "plos", "/Medicine and health sciences/Medical conditions/Muscular dystrophies/Duchenne muscular dystrophy" ], [ "plos", "/Medicine and health sciences/Neurology/Muscular dystrophies/Duchenne muscular dystrophy" ], [ "plos", "/People and places/Geographical locations/Asia/Pakistan" ], [ "plos", "/People and places/Population groupings/Age groups/Children" ], [ "plos", "/People and places/Population groupings/Families/Children" ], [ "plos", "/Research and analysis methods/Research assessment/Systematic reviews" ], [ "plos", "/Social sciences/Psychology/Cognitive psychology/Learning/Human learning" ], [ "plos", "/Social sciences/Psychology/Psychometrics" ] ], "openalex": "W7160429547", "pdf_url": null, "pmcid": null, "pmid": null, "published": "2026-11-06", "publisher": "Public Library of Science", "ris": "https://research.draconitic.com/paper/6bcjd2xs6hm07.ris", "sources": [ { "first_seen": "2026-11-07T12:20:24Z", "native_id": "10.1371/journal.pone.0349543", "source": "plos", "url": "https://doi.org/10.1371/journal.pone.0349543" } ], "subjects": [ { "field": "biology", "origin": "plos:genetics", "subfield": "genetics-genomics" }, { "field": "biology", "origin": "plos:neuroscience", "subfield": "neuroscience" }, { "field": "medicine", "origin": "plos:epidemiology", "subfield": "epidemiology-public-health" } ], "title": "A pre-post single-arm pilot trial of a digital multicomponent intervention for self-efficacy enhancement among online support groups of primary caregivers of children with neuro-muscular disorders: SEE-NMD protocol", "title_text": "A pre-post single-arm pilot trial of a digital multicomponent intervention for self-efficacy enhancement among online support groups of primary caregivers of children with neuro-muscular disorders: SEE-NMD protocol", "type": "peer-reviewed", "updated_at": "2026-11-07T12:20:24Z", "url": "https://research.draconitic.com/paper/6bcjd2xs6hm07", "venue": "PLOS One", "versions": [] }