LAL-D affects patients of all ages with clinical manifestations from infancy through adulthood and may have sudden and unpredictable clinical complications.2,3
Infants with LAL-D can face rapid disease progression over a period of weeks that is typically fatal within a matter of months. The median age of death in these patients is 3.7 months.4 Pediatric and adult patients with LAL-D face substantial disease burden.
Approximately 50% of children and adults with LAL-D progress to fibrosis, cirrhosis, or liver transplant within 3 years of clinical manifestation onset.5 The median age of onset in children and adults with LAL-D is 5.8 years (range 0 to 42 years).6 LAL-D can be diagnosed with a simple blood test.7
Symptoms of LAL-D2,3
- Multi-organ damage
- Cardiovascular disease manifestations including dyslipidemia, accelerated atherosclerosis, coronary artery disease
- Liver damage including fibrosis, cirrhosis, and failure
- Failure to thrive and premature death