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heritable pulmonary arterial hypertension (FPAH) - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

heritable pulmonary arterial hypertension

MONDO:0017148

Also known as:FPAHHPAHfamilial pulmonary arterial hypertension

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Overview

Heritable pulmonary arterial hypertension (HPAH) is a form of pulmonary arterial hypertension (PAH), occurring due to mutations in PAH predisposing genes or in a familial context. HPAH is characterized by elevated pulmonary arterial resistance leading to right heart failure. HPAH is progressive and potentially fatal.

Clinical Trials99 registered · 13 recruiting

Diagnosis

Biomarker and diagnostic research for heritable pulmonary arterial hypertension has been reported in the published literature.

Prognosis

Estimated prevalence: <1 in 1,000,000 (VERY_RARE).

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.

99 clinical trials registered, 13 recruiting. Interventions under study include drug therapy, other interventions, medical devices, and biologic therapy. Pipeline includes 8 PHASE4, 11 PHASE3, 17 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT04778046](https://clinicaltrials.gov/study/NCT04778046)

Data assembled from 4 of 12 sources · Last updated Sep 18, 2026, 3:00 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Financial & Access Support (External Programs)

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Pulmonary Arterial Hypertension Program

Premium/CopayClosedOperated by Good Days

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Program availability and eligibility requirements are set by each foundation. Contact them directly to learn more about your options.

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Medical References

External Resources

Orphanet

European rare disease database

Orphanet:275777

GARD

Genetic and Rare Diseases Info Center

GARD:11914

Questions for Your Care Team

→Are any of the 99 active clinical trials a good fit for my situation?
→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

Research Type	Count	% of Total
Patient case studies	13	33%
Disease patterns and progression	8	21%
Research summaries	5	13%
Laboratory research	5	13%
Testing and diagnosis research	3	8%
Clinical study results	3	8%
Other research	1	3%
New treatment approaches	1	3%

Kisho

heritable pulmonary arterial hypertension

Overview

Diagnosis

Prognosis

Research Landscape

Patient Support

Financial & Access Support (External Programs)

Are you a patient organization for this condition?

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Recent Publications

Latest News & Developments

Exploratory assessment of catestatin and thrombospondin-1 as potential biomarkers of disease severity and treatment response in pulmonary arterial hypertension.

Global disparities in access to randomized controlled trials for pulmonary arterial hypertension.

Early detection, treatment initiation, and long-term health outcomes in patients with connective tissue disease-associated pulmonary arterial hypertension: A systematic literature review and expert consensus.

Lancet Results Show Ralinepag Cut Clinical Worsening by 55 Percent in Pulmonary Arterial Hypertension

Insmed moves closer to $6B opportunity as PAH drug shows long-term durability

heritable pulmonary arterial hypertension

Overview

Diagnosis

Prognosis

Research Landscape

Patient Support

Financial & Access Support (External Programs)

Are you a patient organization for this condition?

Medical References

External Resources

Orphanet

GARD

Subtypes

Questions for Your Care Team

Questions for Your Care Team

Recent Publications

Latest News & Developments

Exploratory assessment of catestatin and thrombospondin-1 as potential biomarkers of disease severity and treatment response in pulmonary arterial hypertension.

Global disparities in access to randomized controlled trials for pulmonary arterial hypertension.

Early detection, treatment initiation, and long-term health outcomes in patients with connective tissue disease-associated pulmonary arterial hypertension: A systematic literature review and expert consensus.

Lancet Results Show Ralinepag Cut Clinical Worsening by 55 Percent in Pulmonary Arterial Hypertension

Insmed moves closer to $6B opportunity as PAH drug shows long-term durability