Disease · MONDO_0008491

stiff-person syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. stiff-person syndrome. Diseases, record MONDO_0008491. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0008491

On this page

Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: stiff-person syndrome; Open Targets
Open Targets ID: MONDO_0008491; Open Targets
Other names: Moersch-Woltman syndrome, Stiff-man syndrome, Stiff-man syndrome (disorder); Open Targets
Disease Ontology: DOID:13366; Disease Ontology
MeSH: D016750; MeSH
NCI Thesaurus: C85170; NCI Thesaurus
Orphanet: ORPHA:3198; Orphanet
Highest stage of its drugs: Approval, for 1 of its 11 drugs, from DailyMed; Open Targets

Drugs11

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
DIAZEPAM	CHEMBL12	Small molecule	Approval	DailyMed	4	0	0	Drugs and targets
EFGARTIGIMOD ALFA	CHEMBL4297551	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	CHEMBL2108981		Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
FILGRASTIM	CHEMBL1201567	Protein	Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets
LENOGRASTIM	CHEMBL2109128	Protein	Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets
MESNA	CHEMBL975	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
METHYLPREDNISOLONE	CHEMBL650	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
PEGFILGRASTIM	CHEMBL1201568	Protein	Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets
RITUXIMAB	CHEMBL1201576	Antibody	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 11

See all 11 drugs

Targets20

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.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Phase 1/2	https://conductscience.org/library/genes/1441	Drugs and targets
FCGRT	IgG receptor FcRn large subunit p51	1	IgG receptor FcRn large subunit p51 antagonist	Phase 2	https://conductscience.org/library/genes/2217	Drugs and targets
GABRA1	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Approval	https://conductscience.org/library/genes/2554	Drugs and targets
GABRA2	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Approval	https://conductscience.org/library/genes/2555	Drugs and targets
GABRA3	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Approval	https://conductscience.org/library/genes/2556	Drugs and targets
GABRA4	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Approval	https://conductscience.org/library/genes/2557	Drugs and targets
GABRA5	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Approval	https://conductscience.org/library/genes/2558	Drugs and targets
GABRA6	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Approval	https://conductscience.org/library/genes/2559	Drugs and targets
GABRB1	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Approval	https://conductscience.org/library/genes/2560	Drugs and targets
GABRB2	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Approval	https://conductscience.org/library/genes/2561	Drugs and targets

1 to 10 of 20

See all 20 targets

Trials2

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 2 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT02282514	Stem Cell Transplantation for Stiff Person Syndrome (SPS)	Phase 1/2	23	2021-01-06	Stiff-Person Syndrome	CYCLOPHOSPHAMIDE, MESNA, METHYLPREDNISOLONE, RITUXIMAB	Its record names the disease	ClinicalTrials.gov
NCT06528392	Efgartigimod for Stiff Person Syndrome (ESPS)	Phase 2	10		Stiff-Person Syndrome	EFGARTIGIMOD ALFA	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials0

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

None in this release.

Papers from its trials1

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it
33318163	RESULT	1

Source PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0008491

GET https://conductscience.org/api/diseases/v1/MONDO_0008491/drugs

API reference For AI assistants