Literature DB >> 22711505

A newly recognized syndrome with characteristic facial features, skeletal dysplasia, and developmental delay.

Wagner A R Baratela1, Michael B Bober, George E Tiller, Ericka Okenfuss, Colleen Ditro, Angela Duker, Deborah Krakow, Deborah L Stabley, Katia Sol-Church, William Mackenzie, Ralph Lachman, Charles I Scott.   

Abstract

We describe a series of seven male patients from six different families with skeletal dysplasia, characteristic facial features, and developmental delay. Skeletal findings include patellar dislocation, short tubular bones, mild metaphyseal changes, brachymetacarpalia with stub thumbs, short femoral necks, shallow acetabular roofs, and platyspondyly. Facial features include: a flattened midface with broad nasal bridge, cleft palate or bifid uvula and synophrys. All of the patients demonstrated pre-school onset of a cognitive developmental delay with a shortened attention span. Some of the cognitive delay was masked by a warm and engaging personality. We posit that these individuals have a newly recognized syndrome characterized by the described features. There is some phenotypic overlap between these patients and Desbuquois dysplasia; however molecular testing demonstrated that this is a distinct disorder. Given the family information available for each patient, we are suspicious that the constellation of findings reported herein could be an X-linked recessive syndrome.
Copyright © 2012 Wiley Periodicals, Inc.

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Year:  2012        PMID: 22711505      PMCID: PMC4164294          DOI: 10.1002/ajmg.a.35445

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


  17 in total

1.  [Bone dysplasia with dwarfism and diffuse skeletal alterations].

Authors:  C Piussan; P Maroteaux; I Castroviejo; B Risbourg
Journal:  Arch Fr Pediatr       Date:  1975 Jun-Jul

2.  Spondyloepimetaphyseal dysplasia with multiple dislocations (Hall type): three further cases and evidence of autosomal dominant inheritance.

Authors:  C M Hall; N H Elcioglu; K D MacDermot; A C Offiah; R M Winter
Journal:  J Med Genet       Date:  2002-09       Impact factor: 6.318

Review 3.  Surgical treatment of cervical kyphosis in Larsen syndrome: report of 3 cases and review of the literature.

Authors:  Hironobu Sakaura; Takashi Matsuoka; Motoki Iwasaki; Kazuo Yonenobu; Hideki Yoshikawa
Journal:  Spine (Phila Pa 1976)       Date:  2007-01-01       Impact factor: 3.468

4.  Multiple congenital dislocations associated with characteristic facial abnormality.

Authors:  L J LARSEN; E R SCHOTTSTAEDT; F C BOST
Journal:  J Pediatr       Date:  1950-10       Impact factor: 4.406

5.  Larsen's syndrome: a skeletal dysplasia with multiple joint dislocations and unusual facies.

Authors:  R J Latta; C B Graham; J Aase; S M Scham; D W Smith
Journal:  J Pediatr       Date:  1971-02       Impact factor: 4.406

Review 6.  Desbuquois syndrome: three further cases and review of the literature.

Authors:  G Gillessen-Kaesbach; P Meinecke; M G Ausems; M Nöthen; B Albrecht; F A Beemer; K Zerres
Journal:  Clin Dysmorphol       Date:  1995-04       Impact factor: 0.816

7.  Desbuquois syndrome.

Authors:  M Le Merrer; I D Young; V Stanescu; P Maroteaux
Journal:  Eur J Pediatr       Date:  1991-09       Impact factor: 3.183

8.  Desbuquois dysplasia, a reevaluation with abnormal and "normal" hands: radiographic manifestations.

Authors:  Laurence Faivre; Valérie Cormier-Daire; Alison M Eliott; Fiona Field; Arnold Munnich; Pierre Maroteaux; Martine Le Merrer; Ralph Lachman
Journal:  Am J Med Genet A       Date:  2004-01-01       Impact factor: 2.802

9.  Clinical and genetic heterogeneity in Desbuquois dysplasia.

Authors:  Laurence Faivre; Martine Le Merrer; Klaus Zerres; Mongi Ben Hariz; Déborah Scheffer; Ian D Young; Pierre Maroteaux; Arnold Munnich; Valérie Cormier-Daire
Journal:  Am J Med Genet A       Date:  2004-07-01       Impact factor: 2.802

Review 10.  Nosology and classification of genetic skeletal disorders: 2010 revision.

Authors:  Matthew L Warman; Valerie Cormier-Daire; Christine Hall; Deborah Krakow; Ralph Lachman; Martine LeMerrer; Geert Mortier; Stefan Mundlos; Gen Nishimura; David L Rimoin; Stephen Robertson; Ravi Savarirayan; David Sillence; Juergen Spranger; Sheila Unger; Bernhard Zabel; Andrea Superti-Furga
Journal:  Am J Med Genet A       Date:  2011-03-15       Impact factor: 2.802

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  3 in total

1.  GGC Repeat Expansion and Exon 1 Methylation of XYLT1 Is a Common Pathogenic Variant in Baratela-Scott Syndrome.

Authors:  Amy J LaCroix; Deborah Stabley; Rebecca Sahraoui; Margaret P Adam; Michele Mehaffey; Kelly Kernan; Candace T Myers; Carrie Fagerstrom; George Anadiotis; Yassmine M Akkari; Katherine M Robbins; Karen W Gripp; Wagner A R Baratela; Michael B Bober; Angela L Duker; Dan Doherty; Jennifer C Dempsey; Daniel G Miller; Martin Kircher; Michael J Bamshad; Deborah A Nickerson; Heather C Mefford; Katia Sol-Church
Journal:  Am J Hum Genet       Date:  2018-12-13       Impact factor: 11.025

2.  PGM3 mutations cause a congenital disorder of glycosylation with severe immunodeficiency and skeletal dysplasia.

Authors:  Asbjørg Stray-Pedersen; Paul H Backe; Hanne S Sorte; Lars Mørkrid; Niti Y Chokshi; Hans Christian Erichsen; Tomasz Gambin; Katja B P Elgstøen; Magnar Bjørås; Marcin W Wlodarski; Marcus Krüger; Shalini N Jhangiani; Donna M Muzny; Ankita Patel; Kimiyo M Raymond; Ghadir S Sasa; Robert A Krance; Caridad A Martinez; Shirley M Abraham; Carsten Speckmann; Stephan Ehl; Patricia Hall; Lisa R Forbes; Else Merckoll; Jostein Westvik; Gen Nishimura; Cecilie F Rustad; Tore G Abrahamsen; Arild Rønnestad; Liv T Osnes; Torstein Egeland; Olaug K Rødningen; Christine R Beck; Eric A Boerwinkle; Richard A Gibbs; James R Lupski; Jordan S Orange; Ekkehart Lausch; I Celine Hanson
Journal:  Am J Hum Genet       Date:  2014-06-12       Impact factor: 11.025

Review 3.  Roles of Chondroitin Sulfate Proteoglycans as Regulators of Skeletal Development.

Authors:  Nancy B Schwartz; Miriam S Domowicz
Journal:  Front Cell Dev Biol       Date:  2022-04-08
  3 in total

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