ArticleMuscle & nerve2019
The myotonic dystrophy experience: a North American cross-sectional study.
Article in Muscle & nerve, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
What it found
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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.
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.
Who cites it
14 citing papers in PubMed.
- Myotonic dystrophy family registry. The patient experience.Journal of neuromuscular diseases · 2026Article
- Exploring the impact of myotonia on daily functioning in myotonic dystrophy: a patient-reported survey.BMC neurology · 2026Article
- Changes in RNA splicing as a surrogate endpoint for myotonic dystrophy Type 1 (DM1) clinical trials.Journal of neuromuscular diseases · 2026Review
- Sexual health in neuromuscular diseases: Neglected challenges revealed by a scoping review.Journal of neuromuscular diseases · 2026Review
- Quantitative Magnetic Resonance Imaging of the Forearm in Myotonic Dystrophy Type 1.Tomography (Ann Arbor, Mich.) · 2025Article
- The blind men and the elephant: recognising the multisystem symptoms of myotonic dystrophy type 1.Orphanet journal of rare diseases · 2025Review
- Article
- Latent factors underlying the symptoms of adult-onset myotonic dystrophy type 1 during the clinical course.Orphanet journal of rare diseases · 2024Article
- Clinical features and genetic spectrum of a multicenter Chinese cohort with myotonic dystrophy type 1.Orphanet journal of rare diseases · 2024Article
- Remote assessment of myotonic dystrophy type 1: A feasibility study.Muscle & nerve · 2022Article
- Article
- Cognitive Deficits, Apathy, and Hypersomnolence Represent the Core Brain Symptoms of Adult-Onset Myotonic Dystrophy Type 1.Frontiers in neurology · 2021Article
- Patient Input to Inform the Development of Central Nervous System Outcome Measures in Myotonic Dystrophy.Therapeutic innovation & regulatory science · 2020Article
- Mixed methods analysis of Health-Related Quality of Life in ambulant individuals affected with RYR1-related myopathies pre-post-N-acetylcysteine therapy.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2020Article
Corrections and comments
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Authors and funding
4 authors.
Funding
Abstract
introductionMyotonic dystrophy (DM) is a chronic, multisystemic, neurological condition. Patients and caregivers are uniquely suited to identify what symptoms are most important and highlight the unmet needs that are most relevant to DM.
methodsWe conducted a North American, cross-sectional study of people with DM type-1, congenital DM, and DM type-2 and their family members. We sent patients and caregivers separate surveys to identify and quantitate the issues of greatest importance, examine the differences between groups, and identify the most important challenges experienced by this population.
results1,180 people with DM and 402 family members/caregivers responded to the surveys. They reported considerable physical and cognitive symptoms, extensive diagnostic delays, and varying clinical phenotypes on the basis of DM type. DISCUSSION: Marked disease burden and numerous unmet needs exist in DM. These needs vary based on DM type and highlight the complex clinical phenotypes of these neurological disorders. Muscle Nerve 59:457-464, 2019.
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