A Rare Case of Dual De Novo Mutations Presenting With Infantile Spasms, Congenital Contractures, and Axial Hypotonia

This report presents the case of a male infant with a complex neonatal course marked by congenital hand and foot deformities, axial hypotonia, and subsequent development of treatment-resistant infantile spasms. Despite extensive initial investigations yielding normal results, including a normal brain magnetic resonance imaging (MRI), whole exome sequencing (WES) revealed two distinct, likely de novo, autosomal dominant mutations: a pathogenic variant in the CACNA1E gene, consistent with Developmental and Epileptic Encephalopathy-69 (DEE69), and a likely pathogenic variant in the FBN1 gene, an incidental finding with significant implications for long-term cardiovascular health. This case highlights the diagnostic power of WES in complex neurodevelopmental disorders and underscores the challenges in managing multifactorial presentations.

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Publication Details

Journal
Cureus
Published
2025-12-04
DOI
https://doi.org/10.7759/cureus.98495
Primary Topic
Genomics and Rare Diseases
Type
article
Field-Weighted Citation Impact
0.00
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article

A Rare Case of Dual De Novo Mutations Presenting With Infantile Spasms, Congenital Contractures, and Axial Hypotonia

Hadi Fakih
Cureus
Genomics and Rare Diseases
article

A Rare Case of Dual De Novo Mutations Presenting With Infantile Spasms, Congenital Contractures, and Axial Hypotonia

Hadi Fakih
article en

Abstract

This report presents the case of a male infant with a complex neonatal course marked by congenital hand and foot deformities, axial hypotonia, and subsequent development of treatment-resistant infantile spasms. Despite extensive initial investigations yielding normal results, including a normal brain magnetic resonance imaging (MRI), whole exome sequencing (WES) revealed two distinct, likely de novo, autosomal dominant mutations: a pathogenic variant in the CACNA1E gene, consistent with Developmental and Epileptic Encephalopathy-69 (DEE69), and a likely pathogenic variant in the FBN1 gene, an incidental finding with significant implications for long-term cardiovascular health. This case highlights the diagnostic power of WES in complex neurodevelopmental disorders and underscores the challenges in managing multifactorial presentations.

CureusVol. 17(12)
Lebanese University (LB), Sheikh Ragheb Harb Hospital (LB)
Openalex Percentile: Top 60%
Genomics and Rare Diseases
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