Literature DB >> 24501761

Intellectual disability, coarse face, relative macrocephaly, and cerebellar hypotrophy in two sisters.

Sérgio B Sousa, Fabiana Ramos, Paula Garcia, Rui P Pais, Catarina Paiva, Philip L Beales, Gudrun E Moore, Jorge M Saraiva, Raoul C M Hennekam.   

Abstract

We report on two Portuguese sisters with a very similar phenotype characterized by severe intellectual disability, absent speech, relative macrocephaly, coarse face, cerebellar hypotrophy, and severe ataxia. Additional common features include increased thickness of the cranial vault, delayed dental eruption, talipes equino-varus, clinodactyly, and camptodactyly of the fifth finger. The older sister has retinal dystrophy and the younger sister has short stature. Their parents are consanguineous. We suggest this condition constitutes a previously unreported autosomal recessive entity.
© 2013 Wiley Periodicals, Inc.

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Year:  2014        PMID: 24501761     DOI: 10.1002/ajmg.a.36235

Source DB:  PubMed          Journal:  Am J Med Genet A        ISSN: 1552-4825            Impact factor:   2.802


  5 in total

Review 1.  Congenital disorders of autophagy: an emerging novel class of inborn errors of neuro-metabolism.

Authors:  Darius Ebrahimi-Fakhari; Afshin Saffari; Lara Wahlster; Jenny Lu; Susan Byrne; Georg F Hoffmann; Heinz Jungbluth; Mustafa Sahin
Journal:  Brain       Date:  2015-12-29       Impact factor: 13.501

2.  SNX14 mutations affect endoplasmic reticulum-associated neutral lipid metabolism in autosomal recessive spinocerebellar ataxia 20.

Authors:  Dale Bryant; Yang Liu; Sanchari Datta; Hanaa Hariri; Marian Seda; Glenn Anderson; Emma Peskett; Charalambos Demetriou; Sergio Sousa; Dagan Jenkins; Peter Clayton; Maria Bitner-Glindzicz; Gudrun E Moore; W Mike Henne; Philip Stanier
Journal:  Hum Mol Genet       Date:  2018-06-01       Impact factor: 6.150

3.  Mutations in SNX14 cause a distinctive autosomal-recessive cerebellar ataxia and intellectual disability syndrome.

Authors:  Anna C Thomas; Hywel Williams; Núria Setó-Salvia; Chiara Bacchelli; Dagan Jenkins; Mary O'Sullivan; Konstantinos Mengrelis; Miho Ishida; Louise Ocaka; Estelle Chanudet; Chela James; Francesco Lescai; Glenn Anderson; Deborah Morrogh; Mina Ryten; Andrew J Duncan; Yun Jin Pai; Jorge M Saraiva; Fabiana Ramos; Bernadette Farren; Dawn Saunders; Bertrand Vernay; Paul Gissen; Anna Straatmaan-Iwanowska; Frank Baas; Nicholas W Wood; Joshua Hersheson; Henry Houlden; Jane Hurst; Richard Scott; Maria Bitner-Glindzicz; Gudrun E Moore; Sérgio B Sousa; Philip Stanier
Journal:  Am J Hum Genet       Date:  2014-11-06       Impact factor: 11.025

4.  Biallelic mutations in SNX14 cause a syndromic form of cerebellar atrophy and lysosome-autophagosome dysfunction.

Authors:  Naiara Akizu; Vincent Cantagrel; Maha S Zaki; Lihadh Al-Gazali; Xin Wang; Rasim Ozgur Rosti; Esra Dikoglu; Antoinette Bernabe Gelot; Basak Rosti; Keith K Vaux; Eric M Scott; Jennifer L Silhavy; Jana Schroth; Brett Copeland; Ashleigh E Schaffer; Philip L S M Gordts; Jeffrey D Esko; Matthew D Buschman; Seth J Field; Gennaro Napolitano; Ghada M Abdel-Salam; R Koksal Ozgul; Mahmut Samil Sagıroglu; Matloob Azam; Samira Ismail; Mona Aglan; Laila Selim; Iman G Mahmoud; Sawsan Abdel-Hadi; Amera El Badawy; Abdelrahim A Sadek; Faezeh Mojahedi; Hulya Kayserili; Amira Masri; Laila Bastaki; Samia Temtamy; Ulrich Müller; Isabelle Desguerre; Jean-Laurent Casanova; Ali Dursun; Murat Gunel; Stacey B Gabriel; Pascale de Lonlay; Joseph G Gleeson
Journal:  Nat Genet       Date:  2015-04-06       Impact factor: 38.330

5.  Two Compound Heterozygous Variants in SNX14 Cause Stereotypies and Dystonia in Autosomal Recessive Spinocerebellar Ataxia 20.

Authors:  Nuno Maia; Gabriela Soares; Cecília Silva; Isabel Marques; Bárbara Rodrigues; Rosário Santos; Manuel Melo-Pires; Arjan Pm de Brouwer; Teresa Temudo; Paula Jorge
Journal:  Front Genet       Date:  2020-09-24       Impact factor: 4.599

  5 in total

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