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In the case of a suspected genetic disease, it is a big challenge to integrate the wide range of symptoms, to select the appropriate diagnostic steps and then to evaluate the results. In this case report, we present the medical history of a boy with congenital heart defects, neurodevelopmental and endocrine disorders. In connection with recurrent, psychomotor developmental delay, detection of minor anomalies and recurrent, severe sepsis since his birth, we started his genetic testing. Multiplex ligation-dependent probe amplification (MLPA-) analysis identified MECP2 duplication syndrome. This genetic syndrome, also called X-linked intellectual disability syndrome Lubs-type, is an X-linked recessive genetic defect. The exact frequency of this genetic disorder is unknown. We know about 200 people in the world, based on literature data. The severity of the clinical symptoms is related to the size of the duplicated chromosome segment. In our patient the affected region, in addition to MECP2, also contained SLC6A8, IDH3G, L1CAM, IRAK1, FLNA, GDI1, DKC1, F8 and VAMP7 genes. These genes may have contributed to the appearance of the diverse clinical picture. We emphasize the pathological role of the reduced cortisol response in the background of recurrent, severe septic conditions. A wide spectrum of genetic testing methods is available, but it depends on the clinician to initiate them. Our main goal with this announcement is to draw attention to these special analyses, which can shorten the diagnostic path, therefore during the next pregnancis the prenatal genetic diagnosis is possible. Orv Hetil. 2025; 166(8): 313–316.
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http://dx.doi.org/10.1556/650.2025.33234 | DOI Listing |
Cureus
August 2025
Pediatric Nephrology, Hospital Pediátrico, Unidade Local de Saúde de Coimbra, Coimbra, PRT.
Introduction Nephrogenic diabetes insipidus (NDI) is a rare condition caused by renal resistance to the action of antidiuretic hormone (ADH) at the level of the distal tubule, resulting in impaired urinary concentration and consequent polyuria. NDI may be hereditary, most commonly X-linked due to AVPR2 gene mutations, or acquired. Objective To characterize the clinical features, management strategies, and outcomes of patients with NDI followed at a tertiary pediatric nephrology center.
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July 2025
Pediatric Department, Royal Medical Services, Queen Rania Children's Hospital, Amman, JOR.
Alpha-thalassemia X-linked intellectual disability syndrome (ATR-X syndrome) is a rare genetic disorder caused by mutations in the gene, typically affecting males and presenting with neurodevelopmental and systemic manifestations. We report, to the best of our knowledge, the first genetically confirmed case of ATR-X syndrome in Jordan, involving a two-and-a-half-year-old male patient who presented with global developmental delay, dysmorphic facies, hypotonia, and bilateral cystic kidneys. Despite persistent microcytic anemia, hemoglobin electrophoresis and PCR for alpha-globin gene deletions were negative.
View Article and Find Full Text PDFProc Natl Acad Sci U S A
September 2025
Ministry of Education Key Laboratory of Protein Science, Beijing Advanced Innovation Center for Structural Biology, School of Life Sciences, Tsinghua University, Beijing 100084, China.
Creatine plays a vital role in cellular energy production and adenosine triphosphate (ATP) homeostasis and has also been identified as a neurotransmitter in the mammalian brain. Creatine is transported into cells by the human creatine transporter (hCRT) (SLC6A8), an Na/Cl-dependent symporter encoded on the X chromosome. Mutations in hCRT cause cerebral creatine deficiency syndrome 1, a neurological disorder marked by intellectual disability, speech delay, and seizures.
View Article and Find Full Text PDFMol Syndromol
May 2025
Division of Child Nutrition and Metabolism, Department of Pediatrics, Van Research and Training Hospital, Van, Turkey.
Introduction: X-linked intellectual disability (XLID) is a highly heterogeneous disease. Apart from Fragile X, other diseases that cause XLID are quite rare. The gene variants cause XLID90.
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