familial chylomicronemia syndrome

ORPHA: 4444901 Treatment Available

Contents

1. Overview
2. Available Treatments
3. Clinical Presentation
4. Classification & Codes

Overview

human disease

Available Treatments (1)

Drug	Form	Status	Countries	Lead Time
olezarsen Orphan Cold Chain	—	FDA Approved (2024)	1	—

Clinical Presentation

Signs and symptoms associated with familial chylomicronemia syndrome, sourced from HPO and Orphanet clinical annotations.

Failure to thriveNausea and vomitingDecreased body weightDepressionHypertriglyceridemiaHyperlipidemiaIncreased circulating chylomicron concentrationLipemia retinalisHepatosplenomegalyAcute pancreatitisEpisodic abdominal painRecurrent pancreatitisEruptive xanthomasHepatic steatosisDementiaDiabetes mellitusJaundicePulmonary embolismMemory impairmentPerianal abscessAbnormal emotional state

Classification & Codes

Orphanet Code

ORPHA:444490

familial chylomicronemia syndrome

Orphanet	`ORPHA:444490`
Treatments	1 drug(s)
Symptoms on record	21 signs
Status	published

Treatment Summary

olezarsen· Orphan

Factual Authority

Last Updated3/20/2026

Clinical DataHPO · Orphanet

Drug DataFDA · EMA · CDSCO