ArticleAnnals of clinical and translational neurology2023
Natural history of Becker muscular dystrophy: a multicenter study of 225 patients.
Article in Annals of clinical and translational neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed, 26 citations in OpenAlex.
- Exploring the Content Validity of Patient-Reported Outcome Measures to Capture the Patient Experience of Becker Muscular Dystrophy.Muscle & nerve · 2026Article
- Effects of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults with Becker muscular dystrophy: results of a phase 1b, open-label study.EBioMedicine · 2026Article
- Cardiomyopathy Associated with Subclinical Becker Muscular Dystrophy in a Patient Presenting with Anesthesia-induced Rhabdomyolysis.Internal medicine (Tokyo, Japan) · 2026Article
- Cardiac MRI in cardiac dystrophinopathy: recommendations on imaging.Open heart · 2026Review
- X-Linked Muscular Dystrophy in a Cat with a Putative Variant in theAnimals : an open access journal from MDPI · 2026Article
- Respiratory function in Becker muscular dystrophy: a comprehensive longitudinal study.Journal of neurology, neurosurgery, and psychiatry · 2026Article
- Metabolic Complications and Ultrasound-Detected Liver Abnormalities in Paediatric Patients With Duchenne Muscular Dystrophy.Pediatric obesity · 2026Article
- Characterization of Gait Kinematics and Muscle Function in Becker Muscular Dystrophy Pigs: a pilot study.bioRxiv : the preprint server for biology · 2025Article
- Ubiquitination-targeted therapies improve BMD iPSC myogenic cell engraftment and dystrophin expressionMolecular therapy. Methods & clinical development · 2025Article
- Longitudinal Changes of Motor Function in Becker Muscular Dystrophy.Neurology. Genetics · 2025Article
- Characterisation of a large, single-centre cohort of patients with Becker muscular dystrophy to inform standardised care guidelines.Journal of neurology · 2025Article
- Article
- The Persistence of Duchenne vs Becker Muscular Dystrophies:Neurology. Genetics · 2025Article
- Clinical Characteristics of Patients With Becker Muscular Dystrophy Having Pathogenic Microvariants or Duplications.Neurology. Genetics · 2025Article
- An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.Methods in molecular biology (Clifton, N.J.) · 2025Review
- Multi-parameter quantitative magnetic resonance imaging for early detecting skeletal muscle involvement and predicting functional decline in children with Becker muscular dystrophy.Pediatric radiology · 2025Article
- Muscular dystrophy patients show low exercise-induced blood flow in muscles with normal strength.Annals of clinical and translational neurology · 2024Article
- Progressive cardiomyopathy with intercalated disc disorganization in a rat model of Becker dystrophy.EMBO reports · 2024Article
- User Perspectives and Psychophysiological Manifestations of Fatigue with Trunk Orthosis for Dystrophinopathy Patients.Bioengineering (Basel, Switzerland) · 2024Article
- Methylphenidate treatment of a Chinese boy with Becker muscular dystrophy combined with attention deficit hyperactivity disorder: a case report.Frontiers in neuroscience · 2024Article
Corrections and comments
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Authors and funding
28 authors at 20 institutions in 1 country.
Funding
Abstract
objectiveBecker muscular dystrophy (BMD) is a milder variant of Duchenne muscular dystrophy (DMD), a lethal X-linked muscular disorder. Here, we aim to investigat the clinical involvement of skeletal, respiratory, cardiac, and central nervous systems in patients with BMD, as well as genotype-phenotype relationships.
methodsThis nationwide cohort study investigated the clinical manifestations and genotype-phenotype relationships in 225 patients with BMD having in-frame deletion from 22 medical centers. The primary outcome was to elucidate the association of genotype with skeletal muscle, respiratory, cardiac, and central nervous system disorders. Descriptive statistics were used to analyze the data.
resultsThe average age of the subjects was 31.5 (range, 1-81) years. Initial symptoms of BMD were muscular (60%), followed by asymptomatic hypercreatine kinasemia (32.4%) and central nervous system disorders (5.3%). Gait disturbance was observed in 53.8% of patients and the average age at wheelchair introduction was 36.5 years. The ventilator introduction rate was 6.7% at an average age of 36.6 years. More than 30% of patients had an abnormal electrocardiogram and approximately 15% had heart failure symptoms. Cardiac function on echocardiography varied significantly among the patients. The frequencies of seizures and intellectual/developmental disability were 8.0% and 16.9%, respectively. Exon 45-47deletion (del) was the most common (22.6%), followed by exon 45-48del (13.1%). Patients with exon 45-49del patients demonstrated severe skeletal muscle damage. Patients with exon 45-47del and exon 45-55del patients did not require ventilator use.
interpretationThe study provides important prognostic information for patients and clinicians to establish therapy plans and to implement preventative medicine.
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