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anti-glomerular basement membrane disease - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

anti-glomerular basement membrane disease

MONDO:0009303

Also known as:Goodpasture Syndromeanti-GBM syndromeanti-glomerular basement membrane antibody disease

Find Clinical TrialsTrials

Overview

An autoimmune disease that affects the lungs and kidneys and is characterized by pulmonary alveolar hemorrhage (bleeding in the lungs) and a kidney disease known as glomerulonephritis. Some use the term 'Goodpasture syndrome' for the findings of glomerulonephritis and pulmonary hemorrhage and the term 'Goodpasture disease' for those patients with glomerulonephritis, pulmonary hemorrhage, and anti-GBM antibodies. Currently, the preferred term for both conditions is “ anti-GBM antibody disease ”. Circulating antibodies are directed against the collagen of the part of the kidney known as the glomerular basement membrane (GBM), resulting in acute or rapidly progressive glomerulonephritis. Antibodies also attack the collagen of the air sacs of the lung (alveoli) resulting in bleeding of the lung (pulmonary hemorrhage). Symptoms may include general body discomfort or pain, bleeding from the nose and/or blood in the urine, respiratory problems, anemia, chest pain, and kidney failure. Anti-GBM disease is thought to result from an environmental insult (smoking, infections, exposure to certain drugs) in a person with genetic susceptibility, such as a specific human leukocyte antigen (HLA) type. Diagnosis is confirmed with the presence of anti-GBM antibody in the blood or in the kidney. The treatment of choice is plasmapheresis in conjunction with prednisone and cyclophosphamide.

GeneticsPolygenic (multifactorial)

TreatmentsNo approved treatments · 1 in pipeline

Clinical Trials

Data assembled from 7 of 12 sources · Last updated Sep 19, 2026, 3:31 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

OMIM

Online Mendelian Inheritance in Man

OMIM:233450

Orphanet

European rare disease database

Orphanet:375

GARD

Genetic and Rare Diseases Info Center

GARD:2551

Questions for Your Care Team

→Are any of the 2 active clinical trials a good fit for my situation?
→What is the current development status of veverimer, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Clinical Presentation

Built from Human Phenotype Ontology (HPO) phenotype annotations and GeneReviews clinical descriptions. Features represent findings documented across known cases — not an exhaustive list of possible symptoms.

Features include very common findings: Low red blood cell count (anemia), Chest pain, Exertional dyspnea, and Pulmonary infiltrates and others; and common findings: Crackles, Pallor, Macroscopic hematuria, and Cylindruria and others. 45 total HPO annotations.

Organ System	Phenotype Count	Example Features
Lungs and breathing	9	Hemosiderin-laden macrophages in bronchoalveolar fluid, Bloody bronchoalveolar lavage fluid, Exertional dyspnea
Kidneys and urinary system	8	Reduced kidney function (renal insufficiency), Macroscopic hematuria, Anti-glomerular basement membrane-antibody positivity
Lab test results	3	Anti-glomerular basement membrane-antibody positivity, Anti-myeloperoxidase antibody positivity, Cytoplasmic antineutrophil antibody positivity
Blood and immune system	3	Low red blood cell count (anemia), Vasculitis, Autoimmunity
Brain and nerves	2	Fatigue, Anti-myeloperoxidase antibody positivity
Bones and joints	2	Joint inflammation (arthritis), Arthralgia
Heart and blood vessels	1	Chest pain
Growth and development	1	Weight loss
Metabolism	1	Fever
Eyes	1	Retinal detachment
Muscles	1	Myalgia

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Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for anti-glomerular basement membrane disease. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for anti-glomerular basement membrane disease, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for anti-glomerular basement membrane disease. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
veverimer	veverimer	Renibus Therapeutics Inc.	2025	—	Designated

Gene therapy approaches for anti-glomerular basement membrane disease have been reported in the published literature.

Clinical Trials

2 trials found

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Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

2 clinical trials registered, 1 recruiting. Interventions under study include drug therapy, other interventions, and procedural interventions. Pipeline includes 1 PHASE2. Research is sponsored by a mix of industry and academic institutions.

124 publications have been identified in PubMed for anti-glomerular basement membrane disease. Research spans Case Report / Case Series (56%), Review / Meta-Analysis (16%), and Epidemiology / Natural History (11%).

Research Type	Count	% of Total
Patient case studies	70	56%
Research summaries	20	16%
Disease patterns and progression	14	11%
Other research	8	6%
Testing and diagnosis research	4	3%
Clinical study results	4	3%
Laboratory research	2	2%
New treatment approaches	2	2%

Recent Publications

Tanaka R (2026). [PMID: 42084625](https://pubmed.ncbi.nlm.nih.gov/42084625/). *Pediatr Nephrol*. [Diagnostic / Biomarker]
Suhlrie A (2026). [PMID: 42006224](https://pubmed.ncbi.nlm.nih.gov/42006224/). *Kidney Int Rep*. [Epidemiology / Natural History]
Takenaka Y (2026). [PMID: 41824186](https://pubmed.ncbi.nlm.nih.gov/41824186/). *CEN Case Rep*. [Case Report / Case Series]
Tyrberg L (2026). [PMID: 41522684](https://pubmed.ncbi.nlm.nih.gov/41522684/). *Clin Kidney J*. [Clinical Trial Publication]
Fernández-Lorente L (2026). [PMID: 41584267](https://pubmed.ncbi.nlm.nih.gov/41584267/). *Kidney Int Rep*. [Other]
Doak W (2026). [PMID: 41886661](https://pubmed.ncbi.nlm.nih.gov/41886661/). *R I Med J (2013)*. [Case Report / Case Series]
Ryan R (2026). [PMID: 41695216](https://pubmed.ncbi.nlm.nih.gov/41695216/). *Kidney Med*. [Case Report / Case Series]
Chew A (2026). [PMID: 41526066](https://pubmed.ncbi.nlm.nih.gov/41526066/). *BMJ Case Rep*. [Case Report / Case Series]
Jordanova E (2026). [PMID: 41864883](https://pubmed.ncbi.nlm.nih.gov/41864883/). *BMC Nephrol*. [Case Report / Case Series]
Kuang H (2026). [PMID: 41798942](https://pubmed.ncbi.nlm.nih.gov/41798942/). *Front Immunol*. [Diagnostic / Biomarker]

Kisho

anti-glomerular basement membrane disease

Overview

Medical References

External Resources

OMIM

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Presentation

Explore clinical features

Diagnosis

Treatment

Orphan Drug Designations

Clinical Trials

Prognosis

Research Landscape

Recent Publications

Latest News & Developments

Subarachnoid hemorrhage and cerebral infarction in anti-glomerular basement membrane disease: a case report and literature review.

Immune-complex-mediated membranoproliferative glomerulonephritis with linear IgG staining resembling atypical anti-glomerular basement membrane disease: a case report.

Anti-glomerular basement membrane nephritis following IgA vasculitis in a child: a diagnostic challenge.

World Trade Center (WTC) Health Program; Petition 026-Anti-Glomerular Basement Membrane (Anti-GBM) Glomerulonephritis; Finding of Insufficient Evidence

anti-glomerular basement membrane disease

Overview

Medical References

External Resources

OMIM

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Presentation

Explore clinical features

Diagnosis

Treatment

Orphan Drug Designations

Clinical Trials

Prognosis

Research Landscape

Recent Publications

Latest News & Developments

Subarachnoid hemorrhage and cerebral infarction in anti-glomerular basement membrane disease: a case report and literature review.

Immune-complex-mediated membranoproliferative glomerulonephritis with linear IgG staining resembling atypical anti-glomerular basement membrane disease: a case report.

Anti-glomerular basement membrane nephritis following IgA vasculitis in a child: a diagnostic challenge.

World Trade Center (WTC) Health Program; Petition 026-Anti-Glomerular Basement Membrane (Anti-GBM) Glomerulonephritis; Finding of Insufficient Evidence