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Dermatomyositis (DM) is a type of idiopathic inflammatory myopathy characterized by evocative skin lesions and symmetrical proximal muscle weakness.
Features include very common findings: Abnormal eyelid morphology, Edema, Autoimmunity, and Limb-girdle muscle weakness and others; and common findings: Weight loss, Difficulty breathing (respiratory insufficiency), Recurrent respiratory infections, and Lung scarring (pulmonary fibrosis) and others. 77 total HPO annotations.
Organ System | Phenotype Count | Example Features |
|---|---|---|
Skin | 15 | Gottron's papules, Heliotrope rash, Papule |
Biomarker and diagnostic research for dermatomyositis has been reported in the published literature.
1 FDA-approved treatment is available for dermatomyositis, including Immune Globulin Intravenous (Human) (Gammagard, Gammagard S/D, Iveegam, approved 1986). An additional 10 compounds hold orphan drug designation.
Brand Name | Generic Name | Mechanism | Approved | Market Status |
|---|---|---|---|---|
Phenotype severity distribution: 10 very common features, 32 common features.
Estimated prevalence: 1-9 in 100,000 (Uncommon).
62 clinical trials registered, 36 recruiting. Interventions under study include other interventions, drug therapy, biologic therapy, and procedural interventions. Pipeline includes 2 PHASE4, 7 PHASE3, 14 PHASE2. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT06569472](https://clinicaltrials.gov/study/NCT06569472) |
Data assembled from 6 of 12 sources · Last updated Sep 19, 2026, 3:00 PM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Lab test results | 13 | Elevated circulating hepatic transaminase concentration, Elevated creatine kinase (muscle enzyme) (elevated circulating creatine kinase concentration), Elevated circulating aldolase concentration |
Lungs and breathing | 7 | Difficulty breathing (respiratory insufficiency), Recurrent respiratory infections, Lung scarring (pulmonary fibrosis) |
Muscles | 7 | Difficulty breathing due to muscle weakness (respiratory insufficiency due to muscle weakness), EMG: myopathic abnormalities, Limb-girdle muscle weakness |
Heart and blood vessels | 5 | Myocardial infarction, Pericarditis, High blood pressure in lung arteries (pulmonary arterial hypertension) |
Blood and immune system | 4 | Recurrent respiratory infections, Autoimmunity, Vasculitis |
Digestive system | 4 | Elevated circulating hepatic transaminase concentration, Difficulty swallowing (dysphagia), Feeding difficulties in infancy |
Brain and nerves | 3 | Fatigue, Dysphonia, Difficulty swallowing (dysphagia) |
Bones and joints | 2 | Arthralgia, Joint inflammation (arthritis) |
Growth and development | 1 | Weight loss |
Arms and legs | 1 | Limb-girdle muscle weakness |
Head and neck | 1 | Facial erythema |
Metabolism | 1 | Fever |
Neoplasm | 1 | Neoplasm |
Bivigam |
Immune Globulin Intravenous (Human) |
— |
2012 |
Available |
Gammaplex | Immune Globulin Intravenous (Human) | — | 2009 | Available |
Octagam | Immune Globulin Intravenous (Human) | — | 2004 | Available |
Flebogamma 5%, Flebogamma 5% Dif | Immune Globulin Intravenous (Human) | — | 2003 | Available |
Carimune, Carimune Nf, Panglobulin, Sandoglobulin | Immune Globulin Intravenous (Human) | — | 2000 | Available |
gammar i.v.,gammar-p i.v. | Immune Globulin Intravenous (Human) | — | 2000 | Available |
Gammagard, Gammagard S/D, Iveegam | Immune Globulin Intravenous (Human) | — | 1986 | Available |
Gamimune | Immune Globulin Intravenous (Human) | — | 1981 | Available |
The following drugs have received orphan drug designation from the FDA for dermatomyositis. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor | Designated | Exclusivity End | Designation Status |
|---|---|---|---|---|---|
brepocitinib | brepocitinib | Priovant Therapeutics, Inc. | 2025 | — | Designated |
begelomab | begelomab | ADIENNE SA | 2021 | — | Designated |
Recombinant humanized monoclonal antibody (immunoglobulin gamma-1 with kappa light chains, IgG1kappa) directed against human soluble cytokine interferon beta. | Recombinant humanized monoclonal antibody (immunoglobulin gamma-1 with kappa light chains, IgG1kappa) directed against human soluble cytokine interferon beta. | Pfizer Inc. | 2020 | — | Designated |
(2S,3R)-N-[(2S)-3-(cyclopent-1-en-1-yl)-1-[(2R)-2-methyloxiran-2-yl]-1-oxopropan-2-yl]-3-hydroxy-3-(4-methoxyphenyl)-2-[(2S)-2-[2-(morpholin-4-yl)acetamido]propanamido]propanamide maleate | (2S,3R)-N-[(2S)-3-(cyclopent-1-en-1-yl)-1-[(2R)-2-methyloxiran-2-yl]-1-oxopropan-2-yl]-3-hydroxy-3-(4-methoxyphenyl)-2-[(2S)-2-[2-(morpholin-4-yl)acetamido]propanamido]propanamide maleate | Kezar Life Sciences, Inc. | 2020 | — | Designated |
humanized anti CD20 monoclonal antibody | humanized anti CD20 monoclonal antibody | Biocon Limited | 2019 | — | Withdrawn |
immune globulin subcutaneous (Human) | immune globulin subcutaneous (Human) | CSL Behring | 2018 | — | Withdrawn |
lenabasum | lenabasum | Corbus Pharmaceuticals, Inc. | 2018 | — | Designated |
sodium thiosulfate | sodium thiosulfate | Hope Pharmaceuticals | 2014 | — | Designated |
siponimod | siponimod | Novartis Pharmaceuticals Corporation | 2014 | — | Withdrawn |
Eculizumab | Eculizumab | Alexion Pharmaceuticals, Inc. | 2000 | — | Withdrawn |
Gene therapy approaches for dermatomyositis have been reported in the published literature.
62 trials found
Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis |
PHASE1 |
Chongqing Precision Biotech Co., Ltd |
RECRUITING |
[NCT06698796](https://clinicaltrials.gov/study/NCT06698796) | A Study to Understand How the Study Medicine Dazukibart Works in People With Idiopathic Inflammatory Myopathies | PHASE3 | Pfizer | RECRUITING |
[NCT06672822](https://clinicaltrials.gov/study/NCT06672822) | Intralesional Injection of STS in Treatment of Calcinosis | PHASE2 | Robyn T. Domsic, MD, MPH | RECRUITING |
[NCT06154252](https://clinicaltrials.gov/study/NCT06154252) | RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy | PHASE2 | Cabaletta Bio | RECRUITING |
[NCT07160205](https://clinicaltrials.gov/study/NCT07160205) | Safety and Efficacy of ULSC on Disease Severity and Steroid Tapering in Participants With Dermatomyositis/ Polymyositis (DM/PM), Also Known as Idiopathic Inflammatory Myopathy (IIM) | PHASE2 | Restem, LLC. | RECRUITING |
291 publications have been identified in PubMed for dermatomyositis. Kisho has analyzed 155 by research type. Research spans Review / Meta-Analysis (28%), Epidemiology / Natural History (21%), and Case Report / Case Series (19%).
Research Type | Count | % of Total |
|---|---|---|
Research summaries | 43 | 28% |
Disease patterns and progression | 33 | 21% |
Patient case studies | 29 | 19% |
Laboratory research | 27 | 17% |
Testing and diagnosis research | 17 | 11% |
Clinical study results | 3 | 2% |
Other research | 2 | 1% |
New treatment approaches | 1 | 1% |
Yang L (2026). [PMID: 40470551](https://pubmed.ncbi.nlm.nih.gov/40470551/). *Clin Exp Rheumatol*. [Epidemiology / Natural History]
He W (2026). [PMID: 40679080](https://pubmed.ncbi.nlm.nih.gov/40679080/). *J Eur Acad Dermatol Venereol*. [Epidemiology / Natural History]
Papadopoulou C (2026). [PMID: 42260535](https://pubmed.ncbi.nlm.nih.gov/42260535/). *Pediatr Rheumatol Online J*. [Review / Meta-Analysis]
Sinnaeve A (2026). [PMID: 41578950](https://pubmed.ncbi.nlm.nih.gov/41578950/). *Rheumatology (Oxford)*. [Epidemiology / Natural History]
Saygin D (2026). [PMID: 41785525](https://pubmed.ncbi.nlm.nih.gov/41785525/). *Semin Arthritis Rheum*. [Basic Science / Preclinical]
Liu H (2026). [PMID: 41176910](https://pubmed.ncbi.nlm.nih.gov/41176910/). *Int Immunopharmacol*. [Basic Science / Preclinical]
Zhang M (2026). [PMID: 41580740](https://pubmed.ncbi.nlm.nih.gov/41580740/). *Arthritis Res Ther*. [Case Report / Case Series]
Kitahara Y (2026). [PMID: 41577954](https://pubmed.ncbi.nlm.nih.gov/41577954/). *Sci Rep*. [Diagnostic / Biomarker]
Sandhu A (2026). [PMID: 41768519](https://pubmed.ncbi.nlm.nih.gov/41768519/). *Arch Intern Med Res*. [Diagnostic / Biomarker]
Uribe-Ruíz NA (2026). [PMID: 41620566](https://pubmed.ncbi.nlm.nih.gov/41620566/). *Clin Rheumatol*. [Review / Meta-Analysis]
AI-curated news mentioning dermatomyositis
Updated Sep 13, 2026
A recent study highlights the diagnostic potential of interferon-stimulated gene 15 (ISG15) muscle expression in dermatomyositis. This research could enhance diagnostic accuracy for this rare autoimmune disease.
Orsini will serve as the specialty pharmacy partner for Priovant's LISRAYA™ (brepocitinib), enhancing patient access to this innovative treatment. The drug was evaluated in the VALOR study, the largest placebo-controlled trial for dermatomyositis, indicating significant advancements in rare disease therapies.
Priovant Therapeutics secures FDA approval for its dual TYK2/JAK1 inhibitor, marking a significant milestone as the first approved treatment for adult dermatomyositis (DM). The approval is supported by data from the largest DM study ever conducted, the Valor trial, allowing use without restrictions based on disease activity or previous treatments.
A case study highlights the recovery of a patient with fulminant anti-nuclear matrix protein 2 antibody-positive dermatomyositis, characterized by massive anasarca and respiratory failure, after prolonged immunosuppression and rehabilitation. This research underscores the potential for recovery in severe cases of this rare autoimmune disease.
A recent case report details a pediatric patient with AQP4+ neuromyelitis optica spectrum disorder (NMOSD), MDA5+ clinically amyopathic dermatomyositis, and secondary hemophagocytic lymphohistiocytosis (sHLH). This study contributes to the understanding of these rare conditions and their clinical presentations.