Phenotype expansion, or double trouble: the combination of congenital disorder of glycosylation type 1i and Joubert syndrome type 17

The co-occurrence of two genetic disorders in a single patient, so called double trouble phenomenon, is a rare clinical scenario that significantly complicates the diagnostic process. This is particularly challenging when both disorders affect the nervous system, leading to overlapping phenotypes. C...

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Main Authors: M. V. Sharova, T. V. Markova, A. L. Chukhrova, O. A. Shchagina, E. L. Dadali
Format: Article
Language:Russian
Published: ABV-press 2025-04-01
Series:Нервно-мышечные болезни
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Online Access:https://nmb.abvpress.ru/jour/article/view/650
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author M. V. Sharova
T. V. Markova
A. L. Chukhrova
O. A. Shchagina
E. L. Dadali
author_facet M. V. Sharova
T. V. Markova
A. L. Chukhrova
O. A. Shchagina
E. L. Dadali
author_sort M. V. Sharova
collection DOAJ
description The co-occurrence of two genetic disorders in a single patient, so called double trouble phenomenon, is a rare clinical scenario that significantly complicates the diagnostic process. This is particularly challenging when both disorders affect the nervous system, leading to overlapping phenotypes. Congenital disorders of glycosylation, including the rare congenital 1i type caused by variants in the ALG2 gene, are characterized by psychomotor delay, microcephaly, seizures, hepatomegaly, and ophthalmological abnormalities. Joubert syndrome, associated with variants in the CPLANE1 gene, presents with brain malformations, severe psychomotor delay, oculomotor apraxia, and respiratory disturbances. In this study, we describe a patient with a rare combination of congenital disorders of glycosylation 1i type and Joubert syndrome type 17, caused by previously unreported variants in the ALG2 and CPLANE1 genes. This case highlights the diagnostic challenges and the need for a comprehensive approach in managing patients with multiple genetic disorders.
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id doaj-art-c5fca32b46a545ffbc819c6e45b6b50a
institution Kabale University
issn 2222-8721
2413-0443
language Russian
publishDate 2025-04-01
publisher ABV-press
record_format Article
series Нервно-мышечные болезни
spelling doaj-art-c5fca32b46a545ffbc819c6e45b6b50a2025-08-20T03:38:16ZrusABV-pressНервно-мышечные болезни2222-87212413-04432025-04-01151677210.17650/2222-8721-2025-15-1-67-72410Phenotype expansion, or double trouble: the combination of congenital disorder of glycosylation type 1i and Joubert syndrome type 17M. V. Sharova0T. V. Markova1A. L. Chukhrova2O. A. Shchagina3E. L. Dadali4Research Centre for Medical Genetics;Research Centre for Medical Genetics;Research Centre for Medical Genetics;Research Centre for Medical Genetics;Research Centre for Medical Genetics;The co-occurrence of two genetic disorders in a single patient, so called double trouble phenomenon, is a rare clinical scenario that significantly complicates the diagnostic process. This is particularly challenging when both disorders affect the nervous system, leading to overlapping phenotypes. Congenital disorders of glycosylation, including the rare congenital 1i type caused by variants in the ALG2 gene, are characterized by psychomotor delay, microcephaly, seizures, hepatomegaly, and ophthalmological abnormalities. Joubert syndrome, associated with variants in the CPLANE1 gene, presents with brain malformations, severe psychomotor delay, oculomotor apraxia, and respiratory disturbances. In this study, we describe a patient with a rare combination of congenital disorders of glycosylation 1i type and Joubert syndrome type 17, caused by previously unreported variants in the ALG2 and CPLANE1 genes. This case highlights the diagnostic challenges and the need for a comprehensive approach in managing patients with multiple genetic disorders.https://nmb.abvpress.ru/jour/article/view/650joubert syndromecongenital disorder of glycosylationalg2cplane1double trouble phenomenon
spellingShingle M. V. Sharova
T. V. Markova
A. L. Chukhrova
O. A. Shchagina
E. L. Dadali
Phenotype expansion, or double trouble: the combination of congenital disorder of glycosylation type 1i and Joubert syndrome type 17
Нервно-мышечные болезни
joubert syndrome
congenital disorder of glycosylation
alg2
cplane1
double trouble phenomenon
title Phenotype expansion, or double trouble: the combination of congenital disorder of glycosylation type 1i and Joubert syndrome type 17
title_full Phenotype expansion, or double trouble: the combination of congenital disorder of glycosylation type 1i and Joubert syndrome type 17
title_fullStr Phenotype expansion, or double trouble: the combination of congenital disorder of glycosylation type 1i and Joubert syndrome type 17
title_full_unstemmed Phenotype expansion, or double trouble: the combination of congenital disorder of glycosylation type 1i and Joubert syndrome type 17
title_short Phenotype expansion, or double trouble: the combination of congenital disorder of glycosylation type 1i and Joubert syndrome type 17
title_sort phenotype expansion or double trouble the combination of congenital disorder of glycosylation type 1i and joubert syndrome type 17
topic joubert syndrome
congenital disorder of glycosylation
alg2
cplane1
double trouble phenomenon
url https://nmb.abvpress.ru/jour/article/view/650
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AT alchukhrova phenotypeexpansionordoubletroublethecombinationofcongenitaldisorderofglycosylationtype1iandjoubertsyndrometype17
AT oashchagina phenotypeexpansionordoubletroublethecombinationofcongenitaldisorderofglycosylationtype1iandjoubertsyndrometype17
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