| Literature DB >> 28462983 |
Alberto Casertano1, Paolo Fontana2, Raoul C Hennekam3, Marco Tartaglia4, Rita Genesio2, Tina Barbaro Dieber5, Lucia Ortega, Lucio Nitsch2, Daniela Melis1.
Abstract
Primrose syndrome is characterized by unusual facial features, macrocephaly, intellectual disability, enlarged, and calcified external ears, sparse body hair, and distal muscle wasting. Nine patients have been described in the literature. The disorder is due to missense mutations in ZBTB20. Here we describe one newly diagnosed 18-month-old patient and provide 10 year follow-up of an earlier reported patient, highlighting the progression and complexity of the disorder. Metabolic studies showed reduced glucose tolerance with prevalence of amino acids and fatty acids catabolism, ketogenesis, and gluconeogenesis, resulting in a Krebs cycle reversion.Entities:
Keywords: ZBTB20; dicarboxylic acids; exome sequencing; overgrowth; primrose
Year: 2017 PMID: 28462983 DOI: 10.1002/ajmg.a.38124
Source DB: PubMed Journal: Am J Med Genet A ISSN: 1552-4825 Impact factor: 2.802