Literature DB >> 21964879

Vici syndrome--a rapidly progressive neurodegenerative disorder with hypopigmentation, immunodeficiency and myopathic changes on muscle biopsy.

Edith Said1, Doriette Soler, Caroline Sewry.   

Abstract

Since its first description by Vici et al. [1988], further reports have continued to broaden the clinical phenotype of this rare multisystem disorder. Main features of agenesis of corpus callosum (ACC), hypopigmentation, immunodeficiency/recurrent infections, cataracts, severe failure to thrive, and profound psychomotor delay have been reported in all cases. An additional feature is the recent evidence for neuromuscular involvement. We describe a female infant with the above core features in whom an initial rapid neurological deterioration and associated transient left ventricular hypertrophy and liver dysfunction was followed by relative clinical stability after ten months of age. This case further underlines the clinical phenotype of Vici syndrome as an early onset neurodegenerative disorder with hypopimentation, recurrent infections and muscle findings indicating myopathic and neurogenic features.
Copyright © 2011 Wiley Periodicals, Inc.

Entities:  

Mesh:

Year:  2011        PMID: 21964879     DOI: 10.1002/ajmg.a.34273

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


  12 in total

Review 1.  Autophagy and human diseases.

Authors:  Peidu Jiang; Noboru Mizushima
Journal:  Cell Res       Date:  2013-12-10       Impact factor: 25.617

Review 2.  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

3.  Role of Epg5 in selective neurodegeneration and Vici syndrome.

Authors:  Yan G Zhao; Hongyu Zhao; Huayu Sun; Hong Zhang
Journal:  Autophagy       Date:  2013-05-14       Impact factor: 16.016

4.  EPG5-Related Vici Syndrome: A Primary Defect of Autophagic Regulation with an Emerging Phenotype Overlapping with Mitochondrial Disorders.

Authors:  Shanti Balasubramaniam; Lisa G Riley; Anand Vasudevan; Mark J Cowley; Velimir Gayevskiy; Carolyn M Sue; Caitlin Edwards; Edward Edkins; Reimar Junckerstorff; C Kiraly-Borri; P Rowe; J Christodoulou
Journal:  JIMD Rep       Date:  2017-11-21

5.  Clinical utility gene card for: Vici Syndrome.

Authors:  Thomas Cullup; Carlo Dionisi-Vici; Ay L Kho; Shu Yau; Shehla Mohammed; Mathias Gautel; Heinz Jungbluth
Journal:  Eur J Hum Genet       Date:  2013-07-10       Impact factor: 4.246

Review 6.  Vici syndrome: a review.

Authors:  Susan Byrne; Carlo Dionisi-Vici; Luke Smith; Mathias Gautel; Heinz Jungbluth
Journal:  Orphanet J Rare Dis       Date:  2016-02-29       Impact factor: 4.123

7.  EPG5-related Vici syndrome: a paradigm of neurodevelopmental disorders with defective autophagy.

Authors:  Susan Byrne; Lara Jansen; Jean-Marie U-King-Im; Ata Siddiqui; Hart G W Lidov; Istvan Bodi; Luke Smith; Rachael Mein; Thomas Cullup; Carlo Dionisi-Vici; Lihadh Al-Gazali; Mohammed Al-Owain; Zandre Bruwer; Khalid Al Thihli; Rana El-Garhy; Kevin M Flanigan; Kandamurugu Manickam; Erik Zmuda; Wesley Banks; Ruth Gershoni-Baruch; Hanna Mandel; Efrat Dagan; Annick Raas-Rothschild; Hila Barash; Francis Filloux; Donnell Creel; Michael Harris; Ada Hamosh; Stefan Kölker; Darius Ebrahimi-Fakhari; Georg F Hoffmann; David Manchester; Philip J Boyer; Adnan Y Manzur; Charles Marques Lourenco; Daniela T Pilz; Arveen Kamath; Prab Prabhakar; Vamshi K Rao; R Curtis Rogers; Monique M Ryan; Natasha J Brown; Catriona A McLean; Edith Said; Ulrike Schara; Anja Stein; Caroline Sewry; Laura Travan; Frits A Wijburg; Martin Zenker; Shehla Mohammed; Manolis Fanto; Mathias Gautel; Heinz Jungbluth
Journal:  Brain       Date:  2016-03       Impact factor: 13.501

8.  Defects in autophagosome-lysosome fusion underlie Vici syndrome, a neurodevelopmental disorder with multisystem involvement.

Authors:  Ikumi Hori; Takanobu Otomo; Mitsuko Nakashima; Fuyuki Miya; Yutaka Negishi; Hideaki Shiraishi; Yutaka Nonoda; Shinichi Magara; Jun Tohyama; Nobuhiko Okamoto; Takeshi Kumagai; Konomi Shimoda; Yoshiya Yukitake; Daigo Kajikawa; Tomohiro Morio; Ayako Hattori; Motoo Nakagawa; Naoki Ando; Ichizo Nishino; Mitsuhiro Kato; Tatsuhiko Tsunoda; Hirotomo Saitsu; Yonehiro Kanemura; Mami Yamasaki; Kenjiro Kosaki; Naomichi Matsumoto; Tamotsu Yoshimori; Shinji Saitoh
Journal:  Sci Rep       Date:  2017-06-14       Impact factor: 4.379

9.  A Saudi Infant with Vici Syndrome: Case Report and Literature Review.

Authors:  Alhussain Alzahrani; Abdulrahman Abdullah Alghamdi; Rahaf Waggass
Journal:  Open Access Maced J Med Sci       Date:  2018-06-13

10.  Recessive mutations in EPG5 cause Vici syndrome, a multisystem disorder with defective autophagy.

Authors:  Thomas Cullup; Ay Lin Kho; Carlo Dionisi-Vici; Birgit Brandmeier; Frances Smith; Zoe Urry; Michael A Simpson; Shu Yau; Enrico Bertini; Verity McClelland; Mohammed Al-Owain; Stefan Koelker; Christian Koerner; Georg F Hoffmann; Frits A Wijburg; Amber E ten Hoedt; R Curtis Rogers; David Manchester; Rie Miyata; Masaharu Hayashi; Elizabeth Said; Doriette Soler; Peter M Kroisel; Christian Windpassinger; Francis M Filloux; Salwa Al-Kaabi; Jozef Hertecant; Miguel Del Campo; Stefan Buk; Istvan Bodi; Hans-Hilmar Goebel; Caroline A Sewry; Stephen Abbs; Shehla Mohammed; Dragana Josifova; Mathias Gautel; Heinz Jungbluth
Journal:  Nat Genet       Date:  2012-12-09       Impact factor: 38.330

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