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Disease
Muscular Diseases
Late-stage therapeutic developmentEmerging research
3
Publications
10
Clinical trials
2024
Latest publication
Current focus
Therapeutic developmentInflammation & immunityMetabolic & lifestyle factors
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Immune Checkpoint Inhibitor Myopathy: The Double-Edged Sword of Cancer Immunotherapy.
Research2024-11-08Neurology
Research2024-02-29Nature communications
Skeletal muscle: A review of molecular structure and function, in health and disease.
Research2019-08-13Wiley interdisciplinary reviews. Systems biology and medicine
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Low
Key developments
- 1 clinical trial expected to report results, the earliest in Q1 2027.
Major developments
Major trial resultsHigh impact
Results posted2026-03-10
Upcoming trial readoutImportant
Results expected Q1 20272026-02-25
Clinical MilestonesViewHide
Results posted
Results expected
- 2026-02-25A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related DiseasesResults expected Q1 2027
Activity timeline3
- 2026-03-10ClinicalA Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label ExtensionResults posted
- 2026-02-25ClinicalA Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related DiseasesResults expected Q1 2027
- 2025-09-10ClinicalA Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) PatientsCompleted
Clinical trials
The current development programme across all trial phases.
Clinical programme
10
2
7
5
Late-stage studies
Recently completed
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Publications over time
20202024
Most influential
Major themes7
- Muscular Diseases3
- Gastrointestinal Microbiome1
- Immune Checkpoint Inhibitors1
- Immunotherapy1
- Models, Biological1
- Muscle, Skeletal1
- Neoplasms1
Leading journals3
- Nature communications1
- Neurology1
- Wiley interdisciplinary reviews. Systems biology and medicine1
Leading researchers8
- Akdag G1
- Baleanu AF1
- Beecher G1
- Bowyer RCE1
- García MP1
- Greig C1
- Harridge SDR1
- Hart D1
Affiliations (unnormalised)6
- Centre for Human & Applied Physiological Sciences1
- Clinical Microbiomics1
- GKT School of Medical Education1
- King's Clinical Trials Unit1
- King's College London1
- MRC-Versus Arthritis Centre for Musculoskeletal Ageing Research1
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
Acquired, familial, and congenital disorders of SKELETAL MUSCLE and SMOOTH MUSCLE.
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.