[en] Background: Kleefstra syndrome (KS) is a rare neurodevelopmental disorder caused by haploinsufficiency of EHMT1; it is characterized by global developmental delay, intellectual disability, hypotonia, distinctive facial features, and variable congenital anomalies. Autistic features, behavioral abnormalities and severe speech impairment are frequently reported. However, molecularly confirmed cases of KS from Africa remain extremely limited, largely due to restricted access to genomic diagnostic infrastructures. Methods: We describe a 15-month-old patient from Rwanda presenting with neonatal hypotonia, global developmental delay, short stature, and characteristic dysmorphic facial features. Comprehensive clinical evaluation was performed, followed by trio-based exome sequencing to identify the underlying genetic cause of this neurodevelopmental disorder. Results: Exome sequencing identified a de novo heterozygous frameshift variant in EHMT1 (NM_024757.5: c.2871dup; p. Phe958Leufs*219), confirming the diagnosis of KS. Conclusions: This report presents the first molecularly confirmed case of KS in Rwanda. It highlights additional clinical features like bilateral 5th toe clinodactyly, short stature and absence of obesity in KS. There is a need to further delineate the study of EHMT1 and investigate the natural history of KS across different populations for optimal patient management and to reduce diagnostic odyssey. The diagnostic utility of exome sequencing for neurodevelopmental disorders needs to be strengthened, with strong emphasis on expanding genomic medicine to help diagnose rare diseases in resource-limited settings.
Disciplines :
Genetics & genetic processes
Author, co-author :
Dukuze, Norbert ; Université de Liège - ULiège > Faculté de Médecine > Doct. scienc. médi. ; Center for Human Genetics, School of Medicine and Pharmacy, College of Medicine and Health Sciences, University of Rwanda, KG 11 Ave Gasabo, Kigali P.O. Box 3286, Rwanda ; Department of Biochemistry, Molecular Biology and Genetics, School of Medicine and Pharmacy, College of Medicine and Health Sciences, University of Rwanda, Kigali P.O. Box 4285, Rwanda
Hitayezu, Janvier ; Department of Pediatrics, University Teaching Hospital of Kigali (CHUK), Kigali P.O. Box 655, Rwanda
Uyisenga, Jeanne Primitive ; Université de Liège - ULiège > GIGA ; Department of Biology, College of Science and Technology, University of Rwanda, Kigali P.O. Box 3900, Rwanda
Uwibambe, Esther ; Center for Human Genetics, School of Medicine and Pharmacy, College of Medicine and Health Sciences, University of Rwanda, KG 11 Ave Gasabo, Kigali P.O. Box 3286, Rwanda
CABERG, Jean-Hubert ; Centre Hospitalier Universitaire de Liège - CHU > > Service de génétique
Dideberg, Vinciane ; Université de Liège - ULiège > Département des sciences biomédicales et précliniques
Bours, Vincent ; Université de Liège - ULiège > GIGA > GIGA Cancer - Human Genetics
Alagbonsi, Abdullateef Isiaka ; Department of Physiology, School of Medicine and Pharmacy, College of Medicine and Health Sciences, University of Rwanda, Kigali P.O. Box 3286, Rwanda
Mutesa, Leon; Center for Human Genetics, School of Medicine and Pharmacy, College of Medicine and Health Sciences, University of Rwanda, KG 11 Ave Gasabo, Kigali P.O. Box 3286, Rwanda ; Department of Biochemistry, Molecular Biology and Genetics, School of Medicine and Pharmacy, College of Medicine and Health Sciences, University of Rwanda, Kigali P.O. Box 4285, Rwanda
Uwineza, Annette ; Center for Human Genetics, School of Medicine and Pharmacy, College of Medicine and Health Sciences, University of Rwanda, KG 11 Ave Gasabo, Kigali P.O. Box 3286, Rwanda ; Department of Biochemistry, Molecular Biology and Genetics, School of Medicine and Pharmacy, College of Medicine and Health Sciences, University of Rwanda, Kigali P.O. Box 4285, Rwanda
Language :
English
Title :
The First Case of Kleefstra Syndrome in a Rwandan Patient with Global Developmental Delay.
Publication date :
07 April 2026
Journal title :
Genes
ISSN :
2073-4425
Publisher :
Multidisciplinary Digital Publishing Institute (MDPI), Switzerland
This work was funded by the European Union (EU)/University of Rwanda Enabel-Kwigira Project, grant number RWA210001-10016. The study was also sponsored by the African Academy of Sciences under its African Research Initiative for Scientific Excellence (ARISE) pilot project 262 program, grant number (DCI-PAN AF/2020/420-028). The funder had no role in the study design, data collection and analysis, decision to publish, or preparation of the manuscript.
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