POTS (Postural Orthostatic Tachycardia Syndrome) — RepurpOS

Disease overview

Alterations

136

Molecular (A)

100

Clinical (B–E)

Direct drugs

Via biomarker

117

Natural agents

Natural products

Merged ranked

137

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature · Pipeline phase 6 · Updated 2026-07-10

Identifiers: MONDO: MONDO:0100242 · ORPHA: ORPHA:140436 · MESH: D054972 · UMLS_CUI: C2931185

Right to Try Relevance

Eligibility: This disease meets the federal and state right-to-try legal standard (21 U.S.C. § 360bbb-0a and Montana SB 535): it lacks disease-modifying therapy with indication-specific FDA approval. Patients with Postural Orthostatic Tachycardia Syndrome may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.

Investigational Agents

Phase 3: 2, Phase 2: 17

Most Advanced Phase

Phase 3

Highest dev. stage

Gene Targets

136

Actionable

Pipeline Compounds (Top 6)

Darapladib, Fenretinide, Compound 2 [Pmid: 22380603], Retinoic Acid Agent, Alprenolol, Amifloxacin

Legal Standard: Right-to-try requires the disease lacks "adequate approved therapy for that specific disease" (not symptom-management drugs). This distinction means a disease with antidepressants available still qualifies if no FDA approval exists for THAT disease indication.

View Right-to-Try Compendium

Alterations

136 total · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Acute myeloid leukemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Astrocytoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
B Acute Lymphoblastic Leukemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ependymoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gastrointestinal desmoid tumor	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Glioblastoma multiforme	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Medulloblastoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Melanoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Meningioma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Nephroblastoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Oligodendroglioma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
T-cell acute lymphoblastic leukemias	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
24 Hour Urinary Catecholamine (DOPA) Excretion During Treatment Normalized to Creatinine	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	LOINC
AARS1 alanyl-tRNA synthetase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ABCC5 ATP binding cassette subfamily C member 5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ACO1 aconitase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ANKRD42 ankyrin repeat domain 42	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
APOD apolipoprotein D	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AREL1 apoptosis resistant E3 ubiquitin protein ligase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
B4GALNT4 beta-1,4-N-acetyl-galactosaminyltransferase 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BAX BCL2 associated X, apoptosis regulator	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BRCA2 BRCA2 DNA repair associated	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BRMS1L BRMS1 like transcriptional repressor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Blood pressure	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
CADM1 cell adhesion molecule 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CAMK2A calcium/calmodulin dependent protein kinase II alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CCHCR1 coiled-coil alpha-helical rod protein 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CERS2 ceramide synthase 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
COG4 component of oligomeric golgi complex 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
COL22A1 collagen type XXII alpha 1 chain	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
COL4A3 collagen type IV alpha 3 chain	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
COL6A5 collagen type VI alpha 5 chain	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CPEB4 cytoplasmic polyadenylation element binding protein 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CREBBP CREB binding lysine acetyltransferase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Change from baseline in quality of life over time	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
Composite Dysautonomia Symptom Score (COMPASS 31)	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
DBN1 drebrin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DHX34 DExH-box helicase 34	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
EGFR epidermal growth factor receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
EIF2B5 eukaryotic translation initiation factor 2B subunit epsilon	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
EQ-5D-5L Quality of Life Score	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
ERBB2 erb-b2 receptor tyrosine kinase 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
End-tidal CO2 levels	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
FAM215A family with sequence similarity 215 member A	Molecular	Gene	—	—	Emerging	Open Targets	Ensembl Open Targets
FANCB FA complementation group B	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FASTKD1 FAST kinase domains 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FBXO11 F-box protein 11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FDX1 ferredoxin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FST follistatin	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Fatigue Severity Scale (FSS)	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
GABARAPL1 GABA type A receptor associated protein like 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GFAP glial fibrillary acidic protein	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GLS glutaminase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GRM2 glutamate metabotropic receptor 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HEPACAM hepatic and glial cell adhesion molecule	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HERC2 HECT and RLD domain containing E3 ubiquitin protein ligase 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HSD17B1 hydroxysteroid 17-beta dehydrogenase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Heart Rate	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
IDH1 isocitrate dehydrogenase (NADP(+)) 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IL17RD interleukin 17 receptor D	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IPO11 importin 11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IPO7 importin 7	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IPO9 importin 9	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IRAG1 inositol 1,4,5-triphosphate receptor associated 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ISOC2 isochorismatase domain containing 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ITGB1BP1 integrin subunit beta 1 binding protein 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KCNT2 potassium sodium-activated channel subfamily T member 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KLLN killin, p53 regulated DNA replication inhibitor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MAP7D3 MAP7 domain containing 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MFSD12 major facilitator superfamily domain containing 12	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MLC1 modulator of VRAC current 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MLDHR mitochondrial lactate dehydrogenase regulator	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MSN moesin	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MTREX Mtr4 exosome RNA helicase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Medical Research Council (MRC) Dyspnoea Scale	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
Morning salivary cortisol levels	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
NECAP2 NECAP endocytosis associated 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NFXL1 nuclear transcription factor, X-box binding like 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NUMA1 nuclear mitotic apparatus protein 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NUP107 nucleoporin 107	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Neuro Quality of Life Score	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
PALS2 protein associated with LIN7 2, MAGUK p55 family member	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PDGFD platelet derived growth factor D	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PEA15 proliferation and apoptosis adaptor protein 15	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PIK3CB phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit beta	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
POLR2F RNA polymerase II, I and III subunit F	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
POT1 protection of telomeres 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PPARG peroxisome proliferator activated receptor gamma	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PPP2R3A protein phosphatase 2 regulatory subunit B''alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSPH phosphoserine phosphatase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSRC1 proline and serine rich coiled-coil 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PTEN phosphatase and tensin homolog	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Plasma Catecholamines (Norepinephrine) After the Last Dose of Placebo or Carbidopa	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Plasma Dopamine	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Plasma HS-CRP levels	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	LOINC
Plasma IL-1 levels	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Plasma IL-10 levels	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Plasma IL-6 levels	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Plasma Norepinephrine	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Plasma Volume	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Post-Exertional Malaise (PEM) Screener	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
RAG1 recombination activating 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
RAPGEF2 Rap guanine nucleotide exchange factor 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
RASAL1 RAS protein activator like 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
RELCH RAB11 binding and LisH domain, coiled-coil and HEAT repeat containing	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
SEC23A SEC23 homolog A, COPII component	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
SMYD2 SET and MYND domain containing 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
SNRPB2 small nuclear ribonucleoprotein polypeptide B2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
SNX32 sorting nexin 32	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
SP4 Sp4 transcription factor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ST13 ST13 Hsp70 interacting protein	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
SUN2 Sad1 and UNC84 domain containing 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
SUPT7L SPT7 like, STAGA complex subunit gamma	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TBC1D14 TBC1 domain family member 14	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TERT telomerase reverse transcriptase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TM2D1 TM2 domain containing 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TNPO2 transportin 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TP53 tumor protein p53	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TRIM28 tripartite motif containing 28	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TTC13 tetratricopeptide repeat domain 13	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TTC38 tetratricopeptide repeat domain 38	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
TXNDC15 thioredoxin domain containing 15	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
UBE2W ubiquitin conjugating enzyme E2 W	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
UBXN11 UBX domain protein 11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
USP30 ubiquitin specific peptidase 30	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
USP43 ubiquitin specific peptidase 43	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Urinary Dopa	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Urinary Dopamine	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Urinary Norepinephrine	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Urinary Sodium	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
VWA1 von Willebrand factor A domain containing 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
WDR3 WD repeat domain 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ZKSCAN3 zinc finger with KRAB and SCAN domains 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ZNF423 zinc finger protein 423	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ZNF746 zinc finger protein 746	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ZNF93 zinc finger protein 93	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug Name	Mechanism	Type
ACETAZOLAMIDE	None	Gene-targeted
ACETYLCYSTEINE		Gene-targeted
ALLOPURINOL		Gene-targeted
AMPHETAMINE	None	Gene-targeted
ARGIPRESSIN		Gene-targeted
ASPARAGINASE		Gene-targeted
ATENOLOL		Gene-targeted
Atenolol	Beta-blocker - HR control	Gene-targeted
BICALUTAMIDE		Gene-targeted
BICALUTAMIDE	None	Gene-targeted
BOSUTINIB		Gene-targeted
CAMPTOTHECIN		Gene-targeted
CANNABIDIOL		Gene-targeted
CARBACHOL	None	Gene-targeted
CARBOPLATIN		Gene-targeted
CARVEDILOL		Gene-targeted
CETUXIMAB		Gene-targeted
CHORIOGONADOTROPIN ALFA		Gene-targeted
CIPROFLOXACIN		Gene-targeted
CIPROFLOXACIN	None	Gene-targeted
CISPLATIN		Gene-targeted
CLOMIPRAMINE	None	Gene-targeted
CURCUMIN		Gene-targeted
CYCLOPHOSPHAMIDE ANHYDROUS		Gene-targeted
Carvedilol	Beta-blocker - vasodilator	Gene-targeted
DACARBAZINE		Gene-targeted
DASATINIB ANHYDROUS		Gene-targeted
DAUNORUBICIN LIPOSOMAL		Gene-targeted
DIAZEPAM	None	Gene-targeted
DICHLORPHENAMIDE	None	Gene-targeted
DIETHYLSTILBESTROL	None	Gene-targeted
DOCETAXEL ANHYDROUS		Gene-targeted
DOXORUBICIN HYDROCHLORIDE		Gene-targeted
ENZALUTAMIDE		Gene-targeted
EPIRUBICIN	None	Gene-targeted
ERYTHROMYCIN		Gene-targeted
ESTRAMUSTINE		Gene-targeted
ETHOXZOLAMIDE	None	Gene-targeted
ETOPOSIDE		Gene-targeted
Ephedrine	Sympathomimetic	Gene-targeted
FLOXURIDINE		Gene-targeted
FLUDARABINE		Gene-targeted
FLUMAZENIL	None	Gene-targeted
FLUOROURACIL		Gene-targeted
Fludrocortisone	Mineralocorticoid - volume expansion	Gene-targeted
GEMCITABINE		Gene-targeted
GLYBURIDE		Gene-targeted
IMIPRAMINE	None	Gene-targeted
INDAPAMIDE	None	Gene-targeted
INDOMETHACIN	None	Gene-targeted
INTERFERON ALFA-2B		Gene-targeted
IRINOTECAN HYDROCHLORIDE		Gene-targeted
ISOSORBIDE DINITRATE	None	Gene-targeted
Ivabradine	HCN channel inhibitor - HR control	Gene-targeted
LEUCOVORIN CALCIUM		Gene-targeted
LEVODOPA		Gene-targeted
MAFENIDE	None	Gene-targeted
MECAMYLAMINE	None	Gene-targeted
MEDROXYPROGESTERONE ACETATE		Gene-targeted
METHAZOLAMIDE	None	Gene-targeted
METHOTREXATE		Gene-targeted
METHOXSALEN	None	Gene-targeted
METOPROLOL	None	Gene-targeted
MIANSERIN	None	Gene-targeted
MITOXANTRONE HYDROCHLORIDE		Gene-targeted
Metoprolol	Beta-blocker - HR control	Gene-targeted
Midodrine	Sympathomimetic - alpha-1 agonist	Gene-targeted
NALIDIXIC ACID	None	Gene-targeted
NALTREXONE HYDROCHLORIDE		Gene-targeted
NEVIRAPINE		Gene-targeted
NICOTINE	None	Gene-targeted
NICOTINE POLACRILEX		Gene-targeted
NIRAPARIB		Gene-targeted
NORFLOXACIN	None	Gene-targeted
OFLOXACIN	None	Gene-targeted
OLAPARIB		Gene-targeted
OXALIPLATIN		Gene-targeted
PEGASPARGASE		Gene-targeted
PHENOBARBITAL		Gene-targeted
PHENYLEPHRINE HYDROCHLORIDE		Gene-targeted
PHENYTOIN	None	Gene-targeted
PRASTERONE		Gene-targeted
PRAVASTATIN SODIUM		Gene-targeted
PRAZOSIN	None	Gene-targeted
PREDNISOLONE		Gene-targeted
PREDNISONE		Gene-targeted
PROCARBAZINE HYDROCHLORIDE		Gene-targeted
Propranolol	Beta-blocker - HR control	Gene-targeted
Pyridostigmine	Acetylcholinesterase inhibitor	Gene-targeted
ROFECOXIB		Gene-targeted
RUCAPARIB		Gene-targeted
SACITUZUMAB GOVITECAN		Gene-targeted
SILDENAFIL	inhibitor	Gene-targeted
SIROLIMUS		Gene-targeted
SIROLIMUS	None	Gene-targeted
SODIUM CHLORIDE		Gene-targeted
SULBACTAM	None	Gene-targeted
TACROLIMUS ANHYDROUS	None	Gene-targeted
TALAZOPARIB		Gene-targeted
TAZOBACTAM	None	Gene-targeted
TEMOZOLOMIDE		Gene-targeted
TESTOSTERONE		Gene-targeted
TRETINOIN		Gene-targeted
TROGLITAZONE	None	Gene-targeted
VERAPAMIL		Gene-targeted
VINORELBINE		Gene-targeted
Verapamil	Calcium channel blocker	Gene-targeted
ZIDOVUDINE		Gene-targeted

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, and merged ranking. Only agents with registry trials or published clinical literature are shown by default.

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ACETYLCYSTEINE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ALLOPURINOL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ARGIPRESSIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ASPARAGINASE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ATENOLOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BOSUTINIB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CAMPTOTHECIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CANNABIDIOL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBOPLATIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CARVEDILOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CETUXIMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CHORIOGONADOTROPIN ALFA Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CURCUMIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DACARBAZINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DASATINIB ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DAUNORUBICIN LIPOSOMAL Repurposing	Small molecule	Approved	Strong	95	—	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	—
DOXORUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ENZALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EPIRUBICIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ERYTHROMYCIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ESTRAMUSTINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ETHOXZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ETOPOSIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FLOXURIDINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FLUDARABINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FLUMAZENIL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOROURACIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
GEMCITABINE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
GLYBURIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits 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	—	ChEMBL	ChEMBL Open Targets
INTERFERON ALFA-2B Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
IRINOTECAN HYDROCHLORIDE 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
LEUCOVORIN CALCIUM Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
LEVODOPA 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
MEDROXYPROGESTERONE ACETATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
METHAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METHOTREXATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
METHOXSALEN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METOPROLOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MIANSERIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MITOXANTRONE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
NALIDIXIC ACID Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
NALTREXONE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
NEVIRAPINE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
NICOTINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
NICOTINE POLACRILEX Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
NIRAPARIB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
NORFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
OFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
OLAPARIB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
OXALIPLATIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
PEGASPARGASE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
PHENOBARBITAL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
PHENYLEPHRINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
PHENYTOIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
PRASTERONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
PRAVASTATIN SODIUM Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
PRAZOSIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
PREDNISOLONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
PREDNISONE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
PROCARBAZINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ROFECOXIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
RUCAPARIB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
SACITUZUMAB GOVITECAN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
SILDENAFIL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
SIROLIMUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
SIROLIMUS Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
SODIUM CHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
SULBACTAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
TACROLIMUS ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
TALAZOPARIB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
TAZOBACTAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
TEMOZOLOMIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
TESTOSTERONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
TRETINOIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
TROGLITAZONE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
VERAPAMIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
VINORELBINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ZIDOVUDINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DARAPLADIB Repurposing	Small molecule	Phase III	Moderate	70	—	ChEMBL	ChEMBL Open Targets
FENRETINIDE Repurposing	Small molecule	Phase III	Moderate	70	—	ChEMBL	ChEMBL Open Targets
COMPOUND 2 [PMID: 22380603] Repurposing	Small molecule	Phase II	Emerging	50	—	DGIdb	—
RETINOIC ACID AGENT Repurposing	Small molecule	Phase II	Emerging	50	—	DGIdb	—
ALPRENOLOL Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
AMIFLOXACIN Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
BROLAMFETAMINE Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
CARZENIDE Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
DIFLOXACIN Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
DIMECAMINE Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
ELLAGIC ACID Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
ENISOPROST Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
FENCAMFAMIN Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
FLEROXACIN Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
IDOXIFENE Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
LYSERGIDE Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
MERAFLOXACIN Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
TENAMFETAMINE Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
ZIDAPAMIDE Repurposing	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets

No therapeutics in this section.

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ACETAZOLAMIDE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ACETYLCYSTEINE Repurposing via ACO1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ALLOPURINOL Repurposing via CCHCR1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
AMPHETAMINE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ARGIPRESSIN Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
ASPARAGINASE Repurposing via CCHCR1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ATENOLOL Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BOSUTINIB Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CAMPTOTHECIN Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
CANNABIDIOL Repurposing via ABCC5	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CARBACHOL Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBOPLATIN Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CARVEDILOL Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
CETUXIMAB Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
CHORIOGONADOTROPIN ALFA Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CLOMIPRAMINE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CURCUMIN Repurposing via CAMK2A	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
DACARBAZINE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
DASATINIB ANHYDROUS Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DAUNORUBICIN LIPOSOMAL Repurposing via ABCC5	Small molecule	Approved	Strong	95	—	DGIdb	—
DIAZEPAM Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
DOXORUBICIN HYDROCHLORIDE Repurposing via ABCC5	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ENZALUTAMIDE Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EPIRUBICIN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ERYTHROMYCIN Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
ESTRAMUSTINE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
ETHOXZOLAMIDE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ETOPOSIDE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
FLOXURIDINE Repurposing via ABCC5	Small molecule	Approved	Strong	95	—	DGIdb	—
FLUDARABINE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
FLUMAZENIL Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOROURACIL Repurposing via ABCC5	Small molecule	Approved	Strong	95	—	DGIdb	—
GEMCITABINE Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
GLYBURIDE Repurposing via ABCC5	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMIPRAMINE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INTERFERON ALFA-2B Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
IRINOTECAN HYDROCHLORIDE Repurposing via ABCC5	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ISOSORBIDE DINITRATE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
LEUCOVORIN CALCIUM Repurposing via ABCC5	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
LEVODOPA Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
MAFENIDE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MECAMYLAMINE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MEDROXYPROGESTERONE ACETATE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
METHAZOLAMIDE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METHOTREXATE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
METHOXSALEN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METOPROLOL Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MIANSERIN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MITOXANTRONE HYDROCHLORIDE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
NALIDIXIC ACID Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
NALTREXONE HYDROCHLORIDE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
NEVIRAPINE Repurposing via CCHCR1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
NICOTINE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
NICOTINE POLACRILEX Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
NIRAPARIB Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
NORFLOXACIN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
OFLOXACIN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
OLAPARIB Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
OXALIPLATIN Repurposing via ABCC5	Small molecule	Approved	Strong	95	—	DGIdb	—
PEGASPARGASE Repurposing via CCHCR1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
PHENOBARBITAL Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
PHENYLEPHRINE HYDROCHLORIDE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
PHENYTOIN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
PRASTERONE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
PRAVASTATIN SODIUM Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
PRAZOSIN Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
PREDNISOLONE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
PREDNISONE Repurposing via CCHCR1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
PROCARBAZINE HYDROCHLORIDE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
ROFECOXIB Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
RUCAPARIB Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
SACITUZUMAB GOVITECAN Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
SILDENAFIL Repurposing via ABCC5	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
SIROLIMUS Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
SIROLIMUS Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
SODIUM CHLORIDE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
SULBACTAM Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
TACROLIMUS ANHYDROUS Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
TALAZOPARIB Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
TAZOBACTAM Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
TEMOZOLOMIDE Repurposing via BRCA2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
TESTOSTERONE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
TRETINOIN Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
TROGLITAZONE Repurposing via AARS1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
VERAPAMIL Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
VINORELBINE Repurposing via BAX	Small molecule	Approved	Strong	95	—	DGIdb	—
ZIDOVUDINE Repurposing via ABCC5	Small molecule	Approved	Strong	95	—	DGIdb	—
DARAPLADIB Repurposing via AARS1	Small molecule	Phase III	Moderate	70	—	ChEMBL	ChEMBL Open Targets
FENRETINIDE Repurposing via AARS1	Small molecule	Phase III	Moderate	70	—	ChEMBL	ChEMBL Open Targets
COMPOUND 2 [PMID: 22380603] Repurposing via ABCC5	Small molecule	Phase II	Emerging	50	—	DGIdb	—
RETINOIC ACID AGENT Repurposing via APOD	Small molecule	Phase II	Emerging	50	—	DGIdb	—
ALPRENOLOL Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
AMIFLOXACIN Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
BROLAMFETAMINE Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
CARZENIDE Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
DIFLOXACIN Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
DIMECAMINE Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
ELLAGIC ACID Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
ENISOPROST Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
FENCAMFAMIN Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
FLEROXACIN Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
IDOXIFENE Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
LYSERGIDE Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
MERAFLOXACIN Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
TENAMFETAMINE Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
ZIDAPAMIDE Repurposing via AARS1	Small molecule	Phase II	Emerging	45	—	ChEMBL	ChEMBL Open Targets
Midodrine Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Pyridostigmine Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Fludrocortisone Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Sildenafil Repurposing via Clinical Trial	Small molecule	Active trials	Strong	90	—		—
Propranolol Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Atenolol Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Carvedilol Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Metoprolol Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Verapamil Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Ivabradine Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Atomoxetine Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—
Ephedrine Repurposing via Clinical Trial	Small molecule	Approved	Strong	90	—		—
Apixaban Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—
Rivaroxaban Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—
Gabapentin Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—
Amitriptyline Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—
Sertraline Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—
Paroxetine Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—
IVIG Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—
Methylphenidate Repurposing via Clinical Trial	Small molecule	Clinical trials	Strong	90	—		—

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.