Fatty Acid Oxidation DisordersNewborn ScreeningRUSP

Carnitine Palmitoyltransferase I Deficiency

CPT-I

CPT-I deficiency limits transport of long-chain fatty acids into mitochondria for oxidation, predisposing to hypoketotic hypoglycemia under fasting stress. The hepatic form is the classic presentation described in screening contexts. Very rare cases benefit from metabolic clinic guidance and illness protocols.

Prevalence
Very rare
Inheritance
Autosomal recessive
Screened at Birth
Yes
On RUSP
Yes (Recommended Uniform Screening Panel)

About This Data

This disorder is part of the Cureledger rare disease data corpus. We are building privacy-preserving natural history datasets and advocacy tools for rare disease communities. Data is maintained by clinical advisors and the patient community.