wild-type transthyretin amyloid
ORPHA: 3300011 Treatment Available
Overview
human disease
Available Treatments (1)
| Drug | Form | Status | Countries | Lead Time |
|---|---|---|---|---|
| Patisiran Orphan Cold Chain | IV infusion, 2 mg/mL | FDA Approved | 6 | — |
Clinical Presentation
Signs and symptoms associated with wild-type transthyretin amyloid, sourced from HPO and Orphanet clinical annotations.
Congestive heart failureHypertrophic cardiomyopathyMyocardial infarctionPleural effusionAbnormal EKGAbnormal pulmonary interstitial morphologyPedal edemaCardiac transthyretin amyloid depositionPulmonary edemaSensory neuropathyChronic diarrheaHepatomegalyIntermittent diarrheaImpaired vibratory sensationGastrointestinal dysmotilityBowel incontinenceElevated circulating alkaline phosphatase concentrationOrthostatic hypotension due to autonomic dysfunctionAtrial fibrillationAutonomic bladder dysfunctionArrhythmiaConstrictive median neuropathyAbnormal autonomic nervous system physiologyDecreased/absent ankle reflexesRenal insufficiencyProteinuriaNephrotic syndromeNephropathyAortic valve stenosisBradycardiaWeight lossSpinal canal stenosis
Classification & Codes
Orphanet Code
ORPHA:330001wild-type transthyretin amyloid
| Orphanet | ORPHA:330001 |
| Treatments | 1 drug(s) |
| Symptoms on record | 32 signs |
| Status | published |
Treatment Summary
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO