Login / Signup

Hemiarhinia caused by a missense variation in SMCHD1: A mild phenotype in the clinical spectrum of Bosma arhinia microphthalmia syndrome.

Nancy Mizue Kokitsu-NakataVinicius Contrucci Dantas SegarraCristiano TonelloMichele Madeira BrandãoNivaldo AlonsoRoseli Maria Zechi-Ceide
Published in: American journal of medical genetics. Part A (2024)
Bosma arhinia microphthalmia syndrome (BAMS, OMIM #603457) is a rare autosomal dominant disorder caused by heterozygous variation in the SMCHD1 gene on chromosome 18p11. Clinically, it is characterized by microphthalmia, absence or hypoplasia of nose, choanal atresia, anosmia, palatal abnormalities, hypogonadotropic hypogonadism, and cryptorchidism. Here we report a Brazilian patient with a likely pathogenic variation in SMCHD1 gene (c.1418A>T; p.Glu473Val) presenting hemiarhinia associated with short stature and hypogonadotropic hypogonadism. Due to the clinical variability of BAMS, we considered that hemiarhinia, without microphthalmia, in the present case, can be considered a mild form of BAMS and could be considered for screening of SMCHD1 gene variation.
Keyphrases
  • copy number
  • case report
  • genome wide
  • genome wide identification
  • replacement therapy
  • early onset
  • dna methylation
  • intellectual disability
  • genome wide analysis
  • growth hormone