Disease · MONDO_0008310

Hutchinson-Gilford progeria syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Hutchinson-Gilford progeria syndrome. Diseases, record MONDO_0008310. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0008310

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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: Hutchinson-Gilford progeria syndrome; Open Targets
Open Targets ID: MONDO_0008310; Open Targets
Other names: HGPS, Hutchinson Gilford syndrome, Hutchinson-Gilford disease, Hutchinson-Gilford progeria, Hutchinson-Gilford syndrome (disorder), Progeria, Progeria (disorder), Progeria syndrome (disorder) [Ambiguous], Syndrome, Hutchinson-Gilford, premature senility syndrome, progeria syndrome; Open Targets
Disease Ontology: DOID:3911; Disease Ontology
NCI Thesaurus: C34951; NCI Thesaurus
Orphanet: ORPHA:740; Orphanet
Highest stage of its drugs: Approval, for 1 of its 6 drugs, from ClinicalTrials.gov, EMA Human Drugs, FDA, PMDA and TTD; Open Targets

Drugs6

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
LONAFARNIB	CHEMBL298734	Small molecule	Approval	ClinicalTrials.gov, EMA Human Drugs, FDA, PMDA and TTD	10	6	6	Drugs and targets
PRAVASTATIN	CHEMBL1144	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets
ZOLEDRONIC ACID	CHEMBL4303669	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets
PROGERININ	CHEMBL1631723	Small molecule	Phase 2	ClinicalTrials.gov and TTD	3	1	1	Drugs and targets
AUTOLOGOUS CORD BLOOD	CHEMBL4594461		Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets
EVEROLIMUS	CHEMBL1908360	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets5

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
FDPS	Farnesyl pyrophosphate synthase	1	Farnesyl diphosphate synthase inhibitor	Phase 2	https://conductscience.org/library/genes/2224	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	1	FK506-binding protein 1A inhibitor	Phase 1/2	https://conductscience.org/library/genes/2280	Drugs and targets
FNTA	Protein farnesyltransferase	1	Protein farnesyltransferase inhibitor	Approval	https://conductscience.org/library/genes/2339	Drugs and targets
FNTB	Protein farnesyltransferase	1	Protein farnesyltransferase inhibitor	Approval	https://conductscience.org/library/genes/2342	Drugs and targets
HMGCR	3-hydroxy-3-methylglutaryl-coenzyme A reductase	1	HMG-CoA reductase inhibitor	Phase 2	https://conductscience.org/library/genes/3156	Drugs and targets

Trials7

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. 1 more is not shown, since the checks do not confirm it.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00425607	Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria	Phase 2	29	2017-05-24	Progeria, Hutchinson-Gilford Syndrome	LONAFARNIB	Its record names the disease	ClinicalTrials.gov
NCT00879034	A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria	Phase 2	5	2017-07-31	Progeria, Hutchinson-Gilford Syndrome	LONAFARNIB, PRAVASTATIN, ZOLEDRONIC ACID	Its record names the disease	ClinicalTrials.gov
NCT00916747	Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria	Phase 2	85		Progeria	LONAFARNIB, PRAVASTATIN, ZOLEDRONIC ACID	Its record names the disease	ClinicalTrials.gov
NCT02579044	Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria	Phase 1/2	80		Progeria	EVEROLIMUS, LONAFARNIB	Its record names the disease	ClinicalTrials.gov
NCT00731016	Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid	Phase 2	15		Hutchinson-Gilford Progeria Syndrome	PRAVASTATIN, ZOLEDRONIC ACID	Its record names the disease	ClinicalTrials.gov
NCT06775041	Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)	Phase 2	10		Hutchinson-Gilford Progeria Syndrome	LONAFARNIB, PROGERININ	Its record names the disease	ClinicalTrials.gov
NCT03895528	Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy				Progeria, HGPS	LONAFARNIB	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 trials11

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	Source
27400896	RESULT	2	PubMed
24795390	RESULT	2	PubMed
23897869	RESULT	1	PubMed
23179651	RESULT	1	PubMed
23012407	RESULT	1	PubMed
22752073	RESULT	1	PubMed
22460337	RESULT	1	PubMed
36919608	DERIVED	2	PubMed
36507973	DERIVED	2	PubMed
24249802	DERIVED	1	PubMed

1 to 10 of 11

See all 11 papers from its trials

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_0008310

GET https://conductscience.org/api/diseases/v1/MONDO_0008310/drugs

API reference For AI assistants