Disease overview

Alterations

194

Molecular (A)

Clinical (B–E)

Direct drugs

Via biomarker

114

Natural agents

Natural products

Merged ranked

162

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, GreenMedInfo, Examine.com · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission

Spontaneous remission in ~10-15% over 2 years without treatment; IBM (inclusion body myositis) is slowly progressive with no effective treatment; death from interstitial lung disease in dermatomyositis in 5-10%

Best-intervention remission

High-dose corticosteroids + IVIG + steroid-sparing agent (methotrexate/azathioprine): clinical remission in 40-60% of DM/PM; IVIG (ProQolity, FDA approved 2021 for DM): 79% response vs 44% placebo (PROPACK trial); rituximab in refractory: 77% response (RIM trial)

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Large for IBM (no effective treatment); moderate for DM/PM

Primary barrier

biological (IBM) + practice (DM/PM undertreated)

Barrier detail: IBM (the most common myopathy in those >60 years) has no effective treatment despite multiple trial failures (bimagrumab, arimoclomol); DM/PM are managed with high-dose steroids first despite substantial side effects and emerging evidence for biologic first-line approaches

Last SoC change: 2021 — IVIG approved specifically for dermatomyositis (PROPACK Phase 3 — first RCT ever positive for any DM treatment); rituximab widely used off-label; corticosteroids as first-line since 1960s; EULAR/ACR classification criteria updated 2017

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0016367

Alterations

Showing 80 of 194 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abdominal pain	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal eosinophil morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal eyelid morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal hair quantity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal pulmonary interstitial morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Acrocyanosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Alopecia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Angina pectoris	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-MDA5 antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-SUMO-activating enzyme antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-SUMO-activating enzyme subunit 1 antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-SUMO-activating enzyme subunit 2 antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-nuclear matrix protein-2 antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Aplasia/Hypoplasia of the skin	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Arrhythmia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Arthralgia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Arthritis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Autoimmunity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Breast carcinoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bundle branch block	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Calcinosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cardiomyopathy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Constipation	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cough	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cutaneous photosensitivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Diffuse reticular or finely nodular infiltrations	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dry skin	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysarthria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysphagia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysphonia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dyspnea	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
EMG abnormality	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
EMG: myopathic abnormalities	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Edema	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating C-reactive protein concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating aldolase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating creatine kinase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating hepatic transaminase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated erythrocyte sedimentation rate	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Erythema	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Facial erythema	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Fatigue	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Feeding difficulties in infancy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Fever	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gastrointestinal hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gastrointestinal stroma tumor	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gottron sign	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gottron's papules	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Heliotrope rash	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hoarse voice	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypotonia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Inflammatory myopathy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Limb-girdle muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Limitation of joint mobility	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Lung adenocarcinoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Mucosal telangiectasiae	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Muscle spasm	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Myalgia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Myocardial infarction	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Myocarditis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Myositis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Neoplasm	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Palpebral edema	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Papule	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pericarditis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Periorbital edema	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Poikiloderma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Proximal muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Pruritus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pulmonary arterial hypertension	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pulmonary fibrosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Raynaud phenomenon	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent respiratory infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Respiratory insufficiency	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Respiratory insufficiency due to muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Restrictive ventilatory defect	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Shawl sign	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Skin rash	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Skin ulcer	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, merged ranking, and clinical trial / literature evidence (top agents)

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ABATACEPT Repurposing	Small molecule	Approved	Strong	95	5 trials 2 Cochrane 1 Meta-analysis 2 Trial pubs 2 RCT View evidence Registry trials NCT04946669 — An Exploratory Study on the Efficacy and Safety of Abatacept in the Treatment of Refractory Dermatomyositis (UNKNOWN) EARLY_PHASE1 NCT02594735 — Abatacept in Juvenile Dermatomyositis (COMPLETED) PHASE4 NCT02971683 — Trial to Evaluate the Efficacy and Safety of Abatacept in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy (COMPLETED) PHASE3 NCT07138898 — Immunosuppressant Management in Rheumatology Patients Undergoing Elective Total Shoulder Arthroplasty (NOT_YET_RECRUITING) PHASE2 NCT01315938 — Abatacept Treatment in Polymyositis and Dermatomyositis (COMPLETED) PHASE2 Published literature Cochrane review Targeted immunosuppressive and immunomodulatory therapies for idiopathic inflammatory myopathies (The Cochrane database of systematic reviews, 2025) Cochrane review A systematic review on biological therapies in juvenile idiopathic inflammatory myopathies: an evidence gap in precision medicine (Clinical and experimental rheumatology, 2022) Clinical trial publication Effect of CTLA4-Ig (abatacept) treatment on T cells and B cells in peripheral blood of patients with polymyositis and dermatomyositis (Scandinavian journal of immunology, 2019) Clinical trial publication Abatacept in the treatment of adult dermatomyositis and polymyositis: a randomised, phase IIb treatment delayed-start trial (Annals of the rheumatic diseases, 2018) CT.gov search · Cochrane · PubMed	DGIdb	—
ACALABRUTINIB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANTI-THYMOCYTE GLOBULIN Repurposing	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT00010335 — Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders (COMPLETED) PHASE1 CT.gov search · Cochrane · PubMed	DGIdb	—
AVACINCAPTAD PEGOL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BELATACEPT Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BELUMOSUDIL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing	Small molecule	Approved	Strong	95	1 Trial pubs 1 RCT View evidence Published literature Clinical trial publication Elective upper-neck versus whole-neck irradiation of the uninvolved neck in patients with nasopharyngeal carcinoma: an open-label, non-inferiority, multicentre, randomised phase 3 trial (The Lancet. Oncology, 2022) CT.gov search · Cochrane · PubMed	DGIdb	—
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOPIDOGREL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CROVALIMAB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
CYCLOSPORINE Repurposing	Small molecule	Approved	Strong	95	4 trials 2 Cochrane 1 Meta-analysis 6 Trial pubs 2 RCT View evidence Registry trials NCT07377058 — RCT of Tocilizumab for Anti-MDA5+DM (RECRUITING) NA NCT07138898 — Immunosuppressant Management in Rheumatology Patients Undergoing Elective Total Shoulder Arthroplasty (NOT_YET_RECRUITING) PHASE2 NCT00323960 — Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis (COMPLETED) PHASE3 NCT03192657 — Basiliximab Treating Interstitial Pneumonia of CADM (UNKNOWN) PHASE2 Published literature Cochrane review Clinically Amyopathic Dermatomyositis: Epidemiology, Clinical Presentation, Pathophysiology, Treatment, and Prognosis: A Systematic Review (Rheumatology and therapy, 2026) Cochrane review Recommendations for the treatment of anti-melanoma differentiation-associated gene 5-positive dermatomyositis-associated rapidly progressive interstitial lung disease (Seminars in arthritis and rheumatism, 2020) Meta-analysis Treatment of idiopathic inflammatory myositis associated interstitial lung disease: A systematic review and meta-analysis (Autoimmunity reviews, 2019) Systematic review A Child with Refractory and Relapsing Anti-3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Myopathy: Case-Based Review (Journal of neuromuscular diseases, 2023) CT.gov search · Cochrane · PubMed	DGIdb	—
DARATUMUMAB Repurposing	Small molecule	Approved	Strong	95	3 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DASATINIB ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DEXAMETHASONE Repurposing	Small molecule	Approved	Strong	95	1 Meta-analysis 1 Trial pubs View evidence Published literature Meta-analysis Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis (The Cochrane database of systematic reviews, 2012) Clinical trial publication Juvenile dermatomyositis (Indian journal of pediatrics, 1996) CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ECULIZUMAB Repurposing	Small molecule	Approved	Strong	95	1 Cochrane 1 Meta-analysis View evidence Published literature Cochrane review Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis (The Cochrane database of systematic reviews, 2012) CT.gov search · Cochrane · PubMed	DGIdb	—
ENTRECTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
EPIRUBICIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ERLOTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ETHOXZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUMAZENIL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOROURACIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FLUTICASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
GEFITINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
GENTAMICIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
IBALIZUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
IBRUTINIB Repurposing	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN Repurposing	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
INDOMETHACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INSULIN, REGULAR, HUMAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ISATUXIMAB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ISOSORBIDE DINITRATE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
LOVASTATIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
MAFENIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MECAMYLAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METHAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METHOXSALEN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METHYLPREDNISOLONE Repurposing	Small molecule	Approved	Strong	95	5 trials 1 Cochrane 5 Trial pubs 1 RCT View evidence Registry trials NCT00004357 — Absorption of Corticosteroids in Children With Juvenile Dermatomyositis (COMPLETED) PHASE2 NCT05509140 — Clinical Analysis of Juvenile Dermatomyositis Patients (COMPLETED) NCT00323960 — Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis (COMPLETED) PHASE3 NCT00651040 — Combined Treatment of Methotrexate + Glucocorticoids Versus Glucocorticoids Alone in Patients With PM and DM (COMPLETED) PHASE3 NCT00504348 — Investigation in Myositis-associated Pneumonitis of Prednisolone And Concomitant Tacrolimus (COMPLETED) PHASE2, PHASE3 Published literature Cochrane review Prevalence and clinical correlates of infection in idiopathic inflammatory myopathies: A systematic review and meta-analysis of 14,548 patients (Autoimmunity reviews, 2026) Systematic review Macrophage activation syndrome in juvenile dermatomyositis: a systematic review (Rheumatology international, 2020) Clinical trial publication The PRINTO evidence-based proposal for glucocorticoids tapering/discontinuation in new onset juvenile dermatomyositis patients (Pediatric rheumatology online journal, 2019) Clinical trial publication Clinical outcome following B cell depletion therapy in eight patients with refractory idiopathic inflammatory myopathy (Clinical and experimental rheumatology, 2008) Clinical trial publication Pharmacokinetic study of oral prednisolone compared with intravenous methylprednisolone in patients with juvenile dermatomyositis (Arthritis and rheumatism, 2008) Clinical trial publication Efficacy of early treatment of severe juvenile dermatomyositis with intravenous methylprednisolone and methotrexate (Clinical rheumatology, 2000) Clinical trial publication The effect of methylprednisolone pulse therapy in polymyositis/dermatomyositis (Advances in experimental medicine and biology, 1999) CT.gov search · Cochrane · PubMed	DGIdb	—
METOPROLOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MIANSERIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Sources	Links
ALDESLEUKIN	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ANIFROLUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BARICITINIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BREPOCITINIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DAZUKIBART	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
HUMAN IMMUNOGLOBULIN G	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LENABASUM	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
METHOTREXATE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
USTEKINUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ABATACEPT	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ANAKINRA	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
APREMILAST	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BAZLITORAN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BELIMUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CORTICOTROPIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CYCLOPHOSPHAMIDE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CYCLOSPORINE	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DAXDILIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DEUCRAVACITINIB	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ECULIZUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
EFGARTIGIMOD ALFA	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
EMPASIPRUBART	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ENPATORAN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
FLUDARABINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
INFLIXIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
INTERFERON ALFA-2B KINOID	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
METHIMAZOLE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
RAVULIZUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
RESECABTAGENE AUTOLEUCEL	Gene therapy	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
RITUXIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
RUXOLITINIB	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
RUXOPRUBART	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
SIFALIMUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
SIPONIMOD	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
SODIUM THIOSULFATE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
TACROLIMUS	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
TOCILIZUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
TOFACITINIB	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ZETOMIPZOMIB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ETANERCEPT	Biologic	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ABATACEPT Repurposing via CD80	Small molecule	Approved	Strong	95	5 trials 2 Cochrane 1 Meta-analysis 2 Trial pubs 2 RCT View evidence Registry trials NCT04946669 — An Exploratory Study on the Efficacy and Safety of Abatacept in the Treatment of Refractory Dermatomyositis (UNKNOWN) EARLY_PHASE1 NCT02594735 — Abatacept in Juvenile Dermatomyositis (COMPLETED) PHASE4 NCT02971683 — Trial to Evaluate the Efficacy and Safety of Abatacept in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy (COMPLETED) PHASE3 NCT07138898 — Immunosuppressant Management in Rheumatology Patients Undergoing Elective Total Shoulder Arthroplasty (NOT_YET_RECRUITING) PHASE2 NCT01315938 — Abatacept Treatment in Polymyositis and Dermatomyositis (COMPLETED) PHASE2 Published literature Cochrane review Targeted immunosuppressive and immunomodulatory therapies for idiopathic inflammatory myopathies (The Cochrane database of systematic reviews, 2025) Cochrane review A systematic review on biological therapies in juvenile idiopathic inflammatory myopathies: an evidence gap in precision medicine (Clinical and experimental rheumatology, 2022) Clinical trial publication Effect of CTLA4-Ig (abatacept) treatment on T cells and B cells in peripheral blood of patients with polymyositis and dermatomyositis (Scandinavian journal of immunology, 2019) Clinical trial publication Abatacept in the treatment of adult dermatomyositis and polymyositis: a randomised, phase IIb treatment delayed-start trial (Annals of the rheumatic diseases, 2018) CT.gov search · Cochrane · PubMed	DGIdb	—
ACALABRUTINIB Repurposing via BLK	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ACETAZOLAMIDE Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMPHETAMINE Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANTI-THYMOCYTE GLOBULIN Repurposing via CD86	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT00010335 — Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders (COMPLETED) PHASE1 CT.gov search · Cochrane · PubMed	DGIdb	—
AVACINCAPTAD PEGOL Repurposing via C5	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BELATACEPT Repurposing via CD80	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BELUMOSUDIL Repurposing via BLK	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CIPROFLOXACIN Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing via CD80	Small molecule	Approved	Strong	95	1 Trial pubs 1 RCT View evidence Published literature Clinical trial publication Elective upper-neck versus whole-neck irradiation of the uninvolved neck in patients with nasopharyngeal carcinoma: an open-label, non-inferiority, multicentre, randomised phase 3 trial (The Lancet. Oncology, 2022) CT.gov search · Cochrane · PubMed	DGIdb	—
CLOMIPRAMINE Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOPIDOGREL Repurposing via CD80	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CROVALIMAB Repurposing via C5	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
CYCLOSPORINE Repurposing via CD80	Small molecule	Approved	Strong	95	4 trials 2 Cochrane 1 Meta-analysis 6 Trial pubs 2 RCT View evidence Registry trials NCT07377058 — RCT of Tocilizumab for Anti-MDA5+DM (RECRUITING) NA NCT07138898 — Immunosuppressant Management in Rheumatology Patients Undergoing Elective Total Shoulder Arthroplasty (NOT_YET_RECRUITING) PHASE2 NCT00323960 — Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis (COMPLETED) PHASE3 NCT03192657 — Basiliximab Treating Interstitial Pneumonia of CADM (UNKNOWN) PHASE2 Published literature Cochrane review Clinically Amyopathic Dermatomyositis: Epidemiology, Clinical Presentation, Pathophysiology, Treatment, and Prognosis: A Systematic Review (Rheumatology and therapy, 2026) Cochrane review Recommendations for the treatment of anti-melanoma differentiation-associated gene 5-positive dermatomyositis-associated rapidly progressive interstitial lung disease (Seminars in arthritis and rheumatism, 2020) Meta-analysis Treatment of idiopathic inflammatory myositis associated interstitial lung disease: A systematic review and meta-analysis (Autoimmunity reviews, 2019) Systematic review A Child with Refractory and Relapsing Anti-3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Myopathy: Case-Based Review (Journal of neuromuscular diseases, 2023) CT.gov search · Cochrane · PubMed	DGIdb	—
DARATUMUMAB Repurposing via CD38	Small molecule	Approved	Strong	95	3 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DASATINIB ANHYDROUS Repurposing via BLK	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DEXAMETHASONE Repurposing via CD86	Small molecule	Approved	Strong	95	1 Meta-analysis 1 Trial pubs View evidence Published literature Meta-analysis Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis (The Cochrane database of systematic reviews, 2012) Clinical trial publication Juvenile dermatomyositis (Indian journal of pediatrics, 1996) CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing via CCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
ECULIZUMAB Repurposing via C5	Small molecule	Approved	Strong	95	1 Cochrane 1 Meta-analysis View evidence Published literature Cochrane review Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis (The Cochrane database of systematic reviews, 2012) CT.gov search · Cochrane · PubMed	DGIdb	—
ENTRECTINIB Repurposing via BLK	Small molecule	Approved	Strong	95	—	DGIdb	—
EPIRUBICIN Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ERLOTINIB Repurposing via BLK	Small molecule	Approved	Strong	95	—	DGIdb	—
ETHOXZOLAMIDE Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUMAZENIL Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOROURACIL Repurposing via CCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
FLUTICASONE Repurposing via CD163	Small molecule	Approved	Strong	95	—	DGIdb	—
GEFITINIB Repurposing via BLK	Small molecule	Approved	Strong	95	—	DGIdb	—
GENTAMICIN Repurposing via CALR	Small molecule	Approved	Strong	95	—	DGIdb	—
IBALIZUMAB Repurposing via CD4	Small molecule	Approved	Strong	95	—	DGIdb	—
IBRUTINIB Repurposing via BLK	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMIPRAMINE Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN Repurposing via CD86	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
INDOMETHACIN Repurposing via ADM	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INSULIN, REGULAR, HUMAN Repurposing via ADM	Small molecule	Approved	Strong	95	—	DGIdb	—

Natural Products

1 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked)

Reference lookups: GreenMedInfo · Examine.com

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
Creatine Natural product Clinical trial publication Trial of Intravenous Immune Globulin in Dermatomyositis Clinical trial publication The Effect of Creatine Supplementation on Muscle Function in Childhood… Clinical trial publication Efficacy and safety of creatine supplementation in juvenile dermatomyos…	Amino acid	Preclinical	Emerging	7	3 trials 24 Trial pubs View evidence Registry trials NCT01217320 — Creatine Supplementation in Pediatric Rheumatology (UNKNOWN) NA NCT04286178 — Exercise, Creatine and Coenzyme Q10 for Childhood Myositis (COMPLETED) NA NCT02267005 — The Effect of Creatine Supplementation on Muscle Function in Childhood Myositis (COMPLETED) NA Published literature Clinical trial publication Trial of Intravenous Immune Globulin in Dermatomyositis (The New England journal of medicine, 2022) Clinical trial publication The Effect of Creatine Supplementation on Muscle Function in Childhood Myositis: A Randomized, Double-blind, Placebo-controlled Feasibility Study (The Journal of rheumatology, 2021) Clinical trial publication Efficacy and safety of creatine supplementation in juvenile dermatomyositis: A randomized, double-blind, placebo-controlled crossover trial (Muscle & nerve, 2016) Clinical trial publication Evidence for metabolic aberrations in asymptomatic persons with type 2 diabetes after initiation of simvastatin therapy (Translational research : the journal of laboratory and clinical medicine, 2015) Clinical trial publication Safety and possible effects of low-intensity resistance training associated with partial blood flow restriction in polymyositis and dermatomyositis (Arthritis research & therapy, 2014) CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.

Inflammatory Myopathies (Dermatomyositis / PM / IBM)

Disease overview

Remission & chronicity

Alterations

Therapeutics

Natural Products

Part of the Open Source Medicine Foundation Network