Literature DB >> 22210667

Orthopaedic aspects of mucopolysaccharidoses.

Klane K White1.   

Abstract

Skeletal abnormalities are an early and prominent feature of most mucopolysaccharide (MPS) disorders, with the degree of skeletal involvement varying between and within MPS subtypes. Most patients exhibit a constellation of radiographic abnormalities known as dysostosis multiplex, consisting of abnormally shaped vertebrae and ribs, enlarged skull, spatulate ribs, hypoplastic epiphyses, thickened diaphyses and bullet-shaped metacarpals. Thoracolumbar kyphosis or the gibbus deformity is often a key diagnostic clue. Also common are hip dysplasia, genu valgum and, later in the course of the disease, spinal cord compression, which can be life-threatening. Short stature is ubiquitous. Treatment of skeletal manifestations usually involves surgical intervention. All patients with MPS should be considered at high risk for surgical intervention requiring anaesthesia because of airway and cardiac disease manifestations. Regular imaging of the cervical, thoracic and lumbar spine, the hips and the lower extremities is recommended for patients with MPS.

Entities:  

Mesh:

Year:  2011        PMID: 22210667     DOI: 10.1093/rheumatology/ker393

Source DB:  PubMed          Journal:  Rheumatology (Oxford)        ISSN: 1462-0324            Impact factor:   7.580


  47 in total

1.  Delayed hypertrophic differentiation of epiphyseal chondrocytes contributes to failed secondary ossification in mucopolysaccharidosis VII dogs.

Authors:  Sun H Peck; Philip J M O'Donnell; Jennifer L Kang; Neil R Malhotra; George R Dodge; Maurizio Pacifici; Eileen M Shore; Mark E Haskins; Lachlan J Smith
Journal:  Mol Genet Metab       Date:  2015-09-26       Impact factor: 4.797

2.  Growth in patients with mucopolysaccharidosis type III (Sanfilippo disease).

Authors:  J de Ruijter; L Broere; M F Mulder; A T van der Ploeg; M E Rubio-Gozalbo; S B Wortmann; G Visser; F A Wijburg
Journal:  J Inherit Metab Dis       Date:  2013-10-31       Impact factor: 4.982

3.  Spinal cord issues in adult patients with MPS: transition of care survey.

Authors:  Kemel A Ghotme; Fernando Alvarado-Gomez; Christina Lampe; Klane K White; Martha Solano-Villareal; Roberto Giugliani; Paul R Harmatz
Journal:  Childs Nerv Syst       Date:  2018-05-27       Impact factor: 1.475

4.  4-year-old boy referring for diffuse joint stiffness and progressive bilateral visual impairment.

Authors:  Vinh Dat-Minh Le; Paolo Simoni
Journal:  Skeletal Radiol       Date:  2019-03       Impact factor: 2.199

5.  The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade.

Authors:  Elizabeth M Xing; Van W Knox; Patricia A O'Donnell; Tracey Sikura; Yuli Liu; Susan Wu; Margret L Casal; Mark E Haskins; Katherine P Ponder
Journal:  Mol Genet Metab       Date:  2013-04-06       Impact factor: 4.797

6.  Adverse Effects of Genistein in a Mucopolysaccharidosis Type I Mouse Model.

Authors:  Sandra D K Kingma; Tom Wagemans; Lodewijk IJlst; Jurgen Seppen; Marion J J Gijbels; Frits A Wijburg; Naomi van Vlies
Journal:  JIMD Rep       Date:  2015-04-09

7.  Elevated TNF-α is associated with pain and physical disability in mucopolysaccharidosis types I, II, and VI.

Authors:  Lynda E Polgreen; Richard K Vehe; Kyle Rudser; Alicia Kunin-Batson; Jeanine Jarnes Utz; Patricia Dickson; Elsa Shapiro; Chester B Whitley
Journal:  Mol Genet Metab       Date:  2016-01-28       Impact factor: 4.797

Review 8.  Mucopolysaccharidoses: overview of neuroimaging manifestations.

Authors:  Manal Nicolas-Jilwan; Moeenaldeen AlSayed
Journal:  Pediatr Radiol       Date:  2018-05-11

9.  Effects of neonatal enzyme replacement therapy and simvastatin treatment on cervical spine disease in mucopolysaccharidosis I dogs.

Authors:  Joseph A Chiaro; Patricia O'Donnell; Eileen M Shore; Neil R Malhotra; Katherine P Ponder; Mark E Haskins; Lachlan J Smith
Journal:  J Bone Miner Res       Date:  2014-12       Impact factor: 6.741

Review 10.  Pathogenesis and treatment of spine disease in the mucopolysaccharidoses.

Authors:  Sun H Peck; Margret L Casal; Neil R Malhotra; Can Ficicioglu; Lachlan J Smith
Journal:  Mol Genet Metab       Date:  2016-06-04       Impact factor: 4.797

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