A case report of an 11-year-old boy with a de novo variant in NOTCH2 and clinical features characteristic of Hajdu-Cheney syndrome is reported, with acroosteolysis of the distal phalanges of the feet and hands, generalized osteoporosis, musculoskeletal and craniofacial alterations, short stature, bowing of long bones, vertebral anomalies, genu recurvatum, hypertrichosis, joint and skin hyperlaxity, atopic dermatitis, megalocorneas, micrognathia and frequent respiratory infections, among others. Treatment is with bisphosphonates in the framework of bone density improvement and with focal vibration therapy for rehabilitation of the musculoskeletal system and gait improvement. The three generalities of this pathology-phenotypic variability, degenerative character and the presence of generalized osteoporosis and acroosteolysis of the distal phalanges-are seen in this case, whose diagnostic confirmation was made by genetic study.
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http://dx.doi.org/10.3390/jcm11175205 | DOI Listing |
Am J Med Genet A
January 2025
Department of Pediatric Genetics, University of Health Sciences, Ankara City Hospital, Ankara, Turkey.
Hajdu-Cheney syndrome (HCS), caused by a heterozygous gain of function variant of the NOTCH2 gene, is a rare skeletal dysplasia. Although the main presentation is acro-osteolysis, osteoporosis, and facial dysmorphism, having a wide range of clinical manifestations creates diagnostic difficulties. Here, a 15-year-old male patient with HCS who had no complaints until this age except for two short bone fractures and one vertebral collapse fracture due to a fall was reported.
View Article and Find Full Text PDFJ Am Soc Nephrol
January 2025
Department of Pharmacology, Tianjin Key Laboratory of Inflammatory Biology, Center for Cardiovascular Diseases, Haihe Laboratory of Cell Ecosystem, Key Laboratory of Immune Microenvironment and Disease (Ministry of Education), The Province and Ministry Co-sponsored Collaborative Innovation Center for Medical Epigenetics, Key Laboratory of Experimental Hematology, School of Basic Medical Sciences, Tianjin Medical University, Tianjin, China.
Background: Notch signaling, a conserved mechanism of cell-to-cell communication, plays a crucial role in regulating cellular processes such as proliferation and differentiation in a context-dependent manner. However, the specific contribution of Notch signaling to the progression of polycystic kidney disease (PKD) remains unclear.
Methods: We investigated the changes in Notch signaling activity (Notch1-4) in the kidneys of autosomal dominant PKD (ADPKD) patients and two ADPKD mouse models (early and late onset).
Bone
February 2025
Department of Orthopaedic Surgery, UConn Health, Farmington, CT, USA; UConn Musculoskeletal Institute, UConn Health, Farmington, CT, USA.
Hajdu Cheney Syndrome (HCS), a monogenic disorder associated with NOTCH2 pathogenic variants, presents with neurological, craniofacial and skeletal abnormalities. Mouse models of the disease exhibit osteopenia. To determine the consequences of a HCS pathogenic variant in human cells, induced pluripotent NCRM1 and NCRM5 stem (iPS) cells harboring a NOTCH2 mutation or null for HES1 alleles were created.
View Article and Find Full Text PDFHand Surg Rehabil
December 2024
Sixth Affiliated Hospital, South China University of Technology, Foshan, Guangdong, 528200, China. Electronic address:
Indian J Anaesth
June 2024
Department of Neuroanesthesiology, Bangur Institute of Neurosciences, Kolkata, West Bengal, India.
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