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paroxysmal nocturnal hemoglobinuria
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Disease · MONDO_0100244

Targets15

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

1 to 15 of 15

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TargetProteinIts drugs that act on itMechanismsHighest stage of those drugsGene in GenesSource
C5Complement C58Complement C5 binding agent; Complement C5 inhibitor; Complement C5 mRNA RNAi inhibitorApprovalhttps://conductscience.org/library/genes/727Drugs and targets
CSF3RGranulocyte colony-stimulating factor receptor3Granulocyte colony stimulating factor receptor agonistPhase 2https://conductscience.org/library/genes/1441Drugs and targets
CFBComplement factor B2Complement factor B inhibitorApprovalhttps://conductscience.org/library/genes/629Drugs and targets
FKBP1APeptidyl-prolyl cis-trans isomerase FKBP1A2FK506-binding protein 1A inhibitorPhase 2https://conductscience.org/library/genes/2280Drugs and targets
C3Complement C31Complement C3 inhibitorApprovalhttps://conductscience.org/library/genes/718Drugs and targets
CD52CAMPATH-1 antigen1CAMPATH-1 antigen inhibitorPhase 1/2https://conductscience.org/library/genes/1043Drugs and targets
CFDComplement factor D1Complement factor D inhibitorApprovalhttps://conductscience.org/library/genes/1675Drugs and targets
DHFRDihydrofolate reductase1Dihydrofolate reductase inhibitorPhase 2https://conductscience.org/library/genes/1719Drugs and targets
IMPDH1Inosine-5'-monophosphate dehydrogenase (IMPDH)1Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorPhase 2https://conductscience.org/library/genes/3614Drugs and targets
IMPDH2Inosine-5'-monophosphate dehydrogenase (IMPDH)1Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorPhase 2https://conductscience.org/library/genes/3615Drugs and targets
LTB4RLeukotriene B4 receptor1Leukotriene B4 receptor inhibitorPhase 3https://conductscience.org/library/genes/1241Drugs and targets
LTB4R2Leukotriene B4 receptor1Leukotriene B4 receptor inhibitorPhase 3https://conductscience.org/library/genes/56413Drugs and targets
MPLThrombopoietin receptor1Thrombopoietin receptor agonistPhase 2/3https://conductscience.org/library/genes/4352Drugs and targets
MS4A1B-lymphocyte antigen CD201B-lymphocyte antigen CD20 binding agentPhase 2https://conductscience.org/library/genes/931Drugs and targets
PPIAPeptidyl-prolyl cis-trans isomerase A1Cyclophilin A modulatorPhase 2https://conductscience.org/library/genes/5478Drugs and targets

diseases-2026-09-18-0.1.0

Sources 6

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ChEMBL, through Open TargetsCC BY-SA 3.0
  • ClinicalTrials.govNo license stated
  • NLM's MeSHNLM's terms
  • Open TargetsCC0 1.0
  • PubMedNLM's terms
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
  • ClinicalTrials.govnone stated
  • NLM's MeSHNLM's
  • Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
  • PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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