Father-to-daughter transmission in late-onset OTC deficiency: an underestimated mechanism of inheritance of an X-linked disease.
Barbara SiriGiorgia OlivieriFrancesca Romana LepriMartin PomsBianca Maria GoffredoAnna CommoneAntonio NovelliJohannes HäberleCarlo Dionisi-ViciPublished in: Orphanet journal of rare diseases (2024)
Our study highlights how the diagnosis and pedigree analysis of late-onset OTCD may represent a real challenge for clinicians. Therefore, the occurrence of paternal transmission in OTCD should not be underestimated, due to the relevant implications for disease inheritance and risk of recurrence.