Primary familial brain calcification (PFBC) is a rare disorder mostly characterized by calcium deposits in the basal ganglia and a wide spectrum of neurologic and psychiatric symptoms, typically inherited as an autosomal dominant trait. Recently, MYORG was reported as the first autosomal recessive causal gene in PFBC patients of Chinese and Middle Eastern origin. Herein, we describe the first PFBC patient of European descent found to carry a novel homozygous MYORG mutation (p.N511Tfs*243). Interestingly, the patient's father, a heterozygous carrier of the same mutation, showed diffuse bilateral cerebral calcifications with no symptoms other than very mild postural tremor.
Primary familial brain calcification caused by a novel homozygous MYORG mutation in a consanguineous Italian family / Eliana Marisa Ramos, ; Roca, Alessandro; Chumchim, Noravit; Deepika Reddy Dokuru, ; Victoria Van Berlo, ; DE MICHELE, Giovanna; Lieto, Maria; Tedeschi, Enrico; DE MICHELE, Giuseppe; Coppola, Giovanni. - In: NEUROGENETICS. - ISSN 1364-6745. - 20:2(2019), pp. 99-102. [10.1007/s10048-019-00571-8]
Primary familial brain calcification caused by a novel homozygous MYORG mutation in a consanguineous Italian family.
Alessandro Roca;Giovanna De Michele;Maria Lieto;Enrico TedeschiWriting – Review & Editing
;Giuseppe De Michele;COPPOLA, GIOVANNI
2019
Abstract
Primary familial brain calcification (PFBC) is a rare disorder mostly characterized by calcium deposits in the basal ganglia and a wide spectrum of neurologic and psychiatric symptoms, typically inherited as an autosomal dominant trait. Recently, MYORG was reported as the first autosomal recessive causal gene in PFBC patients of Chinese and Middle Eastern origin. Herein, we describe the first PFBC patient of European descent found to carry a novel homozygous MYORG mutation (p.N511Tfs*243). Interestingly, the patient's father, a heterozygous carrier of the same mutation, showed diffuse bilateral cerebral calcifications with no symptoms other than very mild postural tremor.File | Dimensione | Formato | |
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