Importance of neurologic and cutaneous signs in the diagnosis of Schimke immuno-osseous dysplasia

Schimke immuno-osseous dysplasia is an autosomal recessive multisystem disorder caused by defects in SWI/SNF-related, matrix-associated, actin-dependent regulator of chromatin, subfamily a-like 1 gene (SMARCAL1). SMARCAL1 product is a helicase that has role in selective cellular proliferation...

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Main Authors: Ayşe İpek Polat, Uluç Yiş, Müge Ayanoğlu, Ayşe Semra Hız, Handan Güleryüz, Tülay Öztürk Atasoy, Cornelius F Boerkoel
Format: Article
Language:English
Published: Hacettepe University Institute of Child Health 2015-10-01
Series:The Turkish Journal of Pediatrics
Online Access:https://turkjpediatr.org/article/view/1276
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author Ayşe İpek Polat
Uluç Yiş
Müge Ayanoğlu
Ayşe Semra Hız
Handan Güleryüz
Tülay Öztürk Atasoy
Cornelius F Boerkoel
author_facet Ayşe İpek Polat
Uluç Yiş
Müge Ayanoğlu
Ayşe Semra Hız
Handan Güleryüz
Tülay Öztürk Atasoy
Cornelius F Boerkoel
author_sort Ayşe İpek Polat
collection DOAJ
description Schimke immuno-osseous dysplasia is an autosomal recessive multisystem disorder caused by defects in SWI/SNF-related, matrix-associated, actin-dependent regulator of chromatin, subfamily a-like 1 gene (SMARCAL1). SMARCAL1 product is a helicase that has role in selective cellular proliferation. The disorder is characterized by spondyloepiphyseal dysplasia with short stature, nephropathy, T cell deficiency, neurologic and cutaneous signs. Patients may have hyperpigmented skin lesions similar to café au lait spots. Symptoms and disease severity in Schimke immuno-osseous dysplasia varies from patient to patient. Genetic, epigenetic and environmental factors play role on the severity of the disease. Here we report on a patient with short stature, steroid resistant nephrotic syndrome and recurrent infections. Cutaneous findings and developmental delay helped us to reach the diagnosis of Schimke immuno-osseous dysplasia. A homozygous missense mutation in SMARCAL1 gene confirmed the clinical diagnosis.
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institution OA Journals
issn 0041-4301
2791-6421
language English
publishDate 2015-10-01
publisher Hacettepe University Institute of Child Health
record_format Article
series The Turkish Journal of Pediatrics
spelling doaj-art-c27e535951774f70bc7bed6e1deace282025-08-20T02:01:53ZengHacettepe University Institute of Child HealthThe Turkish Journal of Pediatrics0041-43012791-64212015-10-01575Importance of neurologic and cutaneous signs in the diagnosis of Schimke immuno-osseous dysplasiaAyşe İpek Polat0Uluç YişMüge AyanoğluAyşe Semra HızHandan GüleryüzTülay Öztürk AtasoyCornelius F BoerkoelDivision of Pediatric Neurology, Department of Pediatrics, Dokuz Eylül University Faculty of Medicine, İzmir, Turkey. ulyis@yahoo.com. Schimke immuno-osseous dysplasia is an autosomal recessive multisystem disorder caused by defects in SWI/SNF-related, matrix-associated, actin-dependent regulator of chromatin, subfamily a-like 1 gene (SMARCAL1). SMARCAL1 product is a helicase that has role in selective cellular proliferation. The disorder is characterized by spondyloepiphyseal dysplasia with short stature, nephropathy, T cell deficiency, neurologic and cutaneous signs. Patients may have hyperpigmented skin lesions similar to café au lait spots. Symptoms and disease severity in Schimke immuno-osseous dysplasia varies from patient to patient. Genetic, epigenetic and environmental factors play role on the severity of the disease. Here we report on a patient with short stature, steroid resistant nephrotic syndrome and recurrent infections. Cutaneous findings and developmental delay helped us to reach the diagnosis of Schimke immuno-osseous dysplasia. A homozygous missense mutation in SMARCAL1 gene confirmed the clinical diagnosis. https://turkjpediatr.org/article/view/1276
spellingShingle Ayşe İpek Polat
Uluç Yiş
Müge Ayanoğlu
Ayşe Semra Hız
Handan Güleryüz
Tülay Öztürk Atasoy
Cornelius F Boerkoel
Importance of neurologic and cutaneous signs in the diagnosis of Schimke immuno-osseous dysplasia
The Turkish Journal of Pediatrics
title Importance of neurologic and cutaneous signs in the diagnosis of Schimke immuno-osseous dysplasia
title_full Importance of neurologic and cutaneous signs in the diagnosis of Schimke immuno-osseous dysplasia
title_fullStr Importance of neurologic and cutaneous signs in the diagnosis of Schimke immuno-osseous dysplasia
title_full_unstemmed Importance of neurologic and cutaneous signs in the diagnosis of Schimke immuno-osseous dysplasia
title_short Importance of neurologic and cutaneous signs in the diagnosis of Schimke immuno-osseous dysplasia
title_sort importance of neurologic and cutaneous signs in the diagnosis of schimke immuno osseous dysplasia
url https://turkjpediatr.org/article/view/1276
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