ReviewActa myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology2019
Current and emerging therapies in Becker muscular dystrophy (BMD).
Review in Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 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.
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Who cites it
22 citing papers in PubMed, 31 citations in OpenAlex.
- An Open Label Exploratory Clinical Trial Evaluating Safety and Tolerability of Once-Weekly Prednisone in Becker and Limb-Girdle Muscular Dystrophy.Journal of neuromuscular diseases · 2022Trial
- Mexico's First Heart Transplant in a Patient with Becker Muscular Dystrophy Reveals an Overlooked Cause of Idiopathic Dilated Cardiomyopathy.Muscles (Basel, Switzerland) · 2026Article
- Meeting report: Translating exercise research in dystrophinopathy to the clinic.Journal of neuromuscular diseases · 2026Article
- Ubiquitination-targeted therapies improve BMD iPSC myogenic cell engraftment and dystrophin expressionMolecular therapy. Methods & clinical development · 2025Article
- Calpainopathy (limb-girdle muscular dystrophy type R1): clinical features, diagnostic approaches, and biotechnological treatment methods.Journal of neuromuscular diseases · 2025Review
- Characterisation of a large, single-centre cohort of patients with Becker muscular dystrophy to inform standardised care guidelines.Journal of neurology · 2025Article
- Advances in Duchenne Muscular Dystrophy: Diagnostic Techniques and Dystrophin Domain Insights.International journal of molecular sciences · 2025Review
- A Systematic Review on the Application of Virtual Reality for Muscular Dystrophy Rehabilitation: Motor Learning Benefits.Life (Basel, Switzerland) · 2024Review
- Characterization of Phenotypic Variability in Becker Muscular Dystrophy for Clinical Practice and Towards Trial Readiness: A Two-Years Follow up Study.Journal of neuromuscular diseases · 2024Article
- The Importance of a PM&R Consultation for Becker Muscular Dystrophy Patients Admitted with Cardiomyopathy.Case reports in cardiology · 2024Article
- Prenatal diagnosis of 1408 foetuses at risk of DMD/BMD by MLPA and Sanger sequencing combined with STR linkage analysis.BMC medical genomics · 2023Article
- Diagnosis and management of Becker muscular dystrophy: the French guidelines.Journal of neurology · 2023Review
- Becker Muscular Dystrophy Accompanied by Anti-HMGCR Antibody-positive Immune-mediated Necrotizing Myopathy.Internal medicine (Tokyo, Japan) · 2023Article
- Development and Validation of a Western Blot Method to Quantify Mini-Dystrophin in Human Skeletal Muscle Biopsies.The AAPS journal · 2022Article
- Article
- Muscle histological changes in a large cohort of patients affected with Becker muscular dystrophy.Acta neuropathologica communications · 2022Article
- Correlation of Serum IGF-1R, VEGF, and ET Levels with Bone Mineral Density in Type 2 Diabetic Mellitus Patients Treated with Metformin PlusEvidence-based complementary and alternative medicine : eCAM · 2022Article
- Review
- Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence.Frontiers in neurology · 2021Review
- Comparison of Carrier andFrontiers in neurology · 2021Article
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Becker muscular dystrophy (BMD) has onset usually in childhood, frequently by 11 years. BMD can present in several ways such as waddling gait, exercise related cramps with or without myoglobinuria. Rarely cardiomyopathy might be the presenting feature. The evolution is variable. BMD is caused by dystrophin deficiency due to inframe deletions, mutations or duplications in dystrophin gene (Xp21.2) We review here the evolution and current therapy presenting a personal series of cases followed for over two decades, with multifactorial treatment regimen. Early treatment includes steroid treatment that has been analized and personalized for each case. Early treatment of cardiomyopathy with ACE inhibitors is recommended and referral for cardiac transplantation is appropriate in severe cases. Management includes multidisciplinary care with physiotherapy to reduce joint contractures and prolong walking. BMD is slowly progressive with phenotypic variability. Despite childhood onset, independent walking is never lost before the third decade. Personalized medicine is required to tailor treatment to individual cases.
Indexed as
Identifiers
31788661PMC6859412W2980461566What OpenQuestion holds
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.