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pure autonomic failure - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

pure autonomic failure

MONDO:0018608

Also known as:Bradbury Eggleston syndromePAFPure dysautonomia

Find Clinical TrialsTrials

Overview

Pure autonomic failure (PAF) is a neurodegenerative disease that affects the sympathetic branch of the autonomous nervous system and that manifests with orthostatic hypotension.

Treatments1 FDA approved

Clinical Trials14 registered · 6 recruiting

Diagnosis

Biomarker and diagnostic research for pure autonomic failure has been reported in the published literature.

Treatment

1 FDA-approved treatment is available for pure autonomic failure, including DROXIDOPA (NORTHERA, approved 2014).

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status
NORTHERA	DROXIDOPA	—	2014	Available

Prognosis

Estimated prevalence: 1-9 in 1,000,000 (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.

14 clinical trials registered, 6 recruiting. Interventions under study include other interventions, drug therapy, and medical devices. Pipeline includes 4 PHASE1, 1 EARLY_PHASE1, 6 NA. Research is primarily sponsored by academic and government institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT03042988](https://clinicaltrials.gov/study/NCT03042988)

Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 9:26 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:441

GARD

Genetic and Rare Diseases Info Center

GARD:10428

Questions for Your Care Team

→What are the benefits and potential side effects of NORTHERA for managing pure autonomic failure?
→Are any of the 14 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
Research summaries	11	26%
Disease patterns and progression	8	19%
Testing and diagnosis research	7	16%
Laboratory research	7	16%
Other research	4	9%
Patient case studies	4	9%
Clinical study results	2	5%

pure autonomic failure

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

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Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

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