ArticleCells2020
The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle.
Article in Cells, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 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
47 citing papers in PubMed, 58 citations in OpenAlex.
- Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy.Scientific reports · 2022Trial
- Age-dependent musculoskeletal changes during mechanical unloading with bisphosphonate treatment.Bone · 2026Article
- A Biomimetic 3D Human Skeletal Muscle Microtissue for Modeling Biological and Functional Hallmarks of Aging.Small science · 2026Article
- Active immunization against myostatin and activin A improves skeletal muscle performance in growth hormone-deficient mice.GeroScience · 2026Article
- Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation.Cells · 2026Review
- CIRRHOSIS-ASSOCIATED SARCOPENIA: INTEGRATING IMAGING, FUNCTION, AND METABOLISM FOR CLINICAL DECISION-MAKING.Arquivos de gastroenterologia · 2026Review
- ACE-031, a soluble activin type IIB receptor, increases muscle mass and strength in the common marmoset (Callithrix jacchus).PloS one · 2026Article
- From mechanisms to management: a comprehensive review of sarcopenia in gastric cancer.Frontiers in nutrition · 2026Review
- The current clinical management of muscle wasting in hemodialysis patients with end-stage renal disease.International urology and nephrology · 2025Review
- ACE-031, a Soluble Activin Type IIB Receptor, Increases Muscle Mass and Strength in the Common Marmoset (Callithrix jacchus).bioRxiv : the preprint server for biology · 2025Article
- Impact of myokines on chronic liver diseases: exploring the effects of metabolic dysfunction-associated steatotic liver disease (MASLD) on skeletal muscle. A narrative review.European journal of translational myology · 2025Article
- Metabolic crosstalk between skeletal muscle and cartilage tissue: insights into myokines in osteoarthritis.Molecular biology reports · 2025Review
- Relationship Between High Serum Levels of Follistatin with Impaired Physical Function, and Severe Disease Activity in Rheumatoid Arthritis.International journal of molecular sciences · 2025Article
- Myostatin Levels in SMA Following Disease-Modifying Treatments: A Multi-Center Study.Annals of clinical and translational neurology · 2025Article
- Aging: A struggle for beneficial to overcome negative factors made by muscle and bone.Mechanisms of ageing and development · 2025Review
- The Function of Myostatin in Ameliorating Bone Metabolism Abnormalities in Individuals with Type 2 Diabetes Mellitus by Exercise.Current issues in molecular biology · 2025Review
- 30 Years Since the Proposal of Exon Skipping Therapy for Duchenne Muscular Dystrophy and the Future of Pseudoexon Skipping.International journal of molecular sciences · 2025Review
- Associations of circulating apolipoprotein J and myostatin with sarcopenia in older adults with and without type 2 diabetes: a cross-sectional study.Frontiers in endocrinology · 2025Article
- A review of myostatin gene mutations: Enhancing meat production and potential in livestock genetic selection.Open veterinary journal · 2024Review
- Impact of Disease Severity and Disease-Modifying Therapies on Myostatin Levels in SMA Patients.International journal of molecular sciences · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Myostatin inhibition therapy has held much promise for the treatment of muscle wasting disorders. This is particularly true for the fatal myopathy, Duchenne Muscular Dystrophy (DMD). Following on from promising pre-clinical data in dystrophin-deficient mice and dogs, several clinical trials were initiated in DMD patients using different modality myostatin inhibition therapies. All failed to show modification of disease course as dictated by the primary and secondary outcome measures selected: the myostatin inhibition story, thus far, is a failed clinical story. These trials have recently been extensively reviewed and reasons why pre-clinical data collected in animal models have failed to translate into clinical benefit to patients have been purported. However, the biological mechanisms underlying translational failure need to be examined to ensure future myostatin inhibitor development endeavors do not meet with the same fate. Here, we explore the biology which could explain the failed translation of myostatin inhibitors in the treatment of DMD.
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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.