Late-Onset Molybdenum Cofactor Deficiency Type A: A Treatable Cause of Developmental Delay.

Lund, Allan M; Berland, Siren; Tangeraas, Trine; Christensen, Mette; Confer, Nils; Squires, Liza; Brannsether, Bente · Pediatrics · 2024

case_report · Level V

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Abstract

Molybdenum cofactor deficiency classically presents in neonates with intractable seizures; however, milder cases generally present before age 2 years with developmental delays and may go undiagnosed. Early diagnosis, and safe, US Food and Drug Administration-approved substrate replacement are critical to preserve neurologic function. This article discusses 2 children who presented with late-onset molybdenum cofactor deficiency type A.

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