Novel VIPAS39 mutation in a syndromic patient with arthrogryposis, renal tubular dysfunction and intrahepatic cholestasis.

نویسندگان

  • Majid Aflatounian
  • Holly Smith
  • Fatemeh Farahani
  • Azam Tofighi Naeem
  • Anna Straatman-Iwanowska
  • Samaneh Zoghi
  • Urvi Khatri
  • Parisa Tajdini
  • Gholam Hossein Fallahi
  • Paul Gissen
  • Nima Rezaei
چکیده

ARC syndrome is a rare autosomal recessive disease, characterized by arthrogryposis, renal tubular dysfunction and cholestasis. Herein a 2.5 month old infant with dysmorphic features, including small anterior fontanel, low set ears, beaked nose and high arched palate is presented who was referred because of icterus. He also suffered from some additional anomalies, including unilateral choanal atresia, club foot, and bilateral developmental dislocation of hip, while further studies showed renal tubular acidosis and hearing impairment in addition to cholestasis. Genetic studies showed a homozygous mutation in the VIPAS39 gene. Making the definite diagnosis of the syndrome is important, while increased risk of mutation in other siblings highlights the importance of prenatal diagnosis.

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عنوان ژورنال:
  • European journal of medical genetics

دوره 59 4  شماره 

صفحات  -

تاریخ انتشار 2016