In a 3-year-old boy with short stature, developmental delay, and dry skin, steroid sulphatase deficiency and a submicroscopic terminal deletion of Xp were found. Except for the short stature, no major clinical signs of X-linked recessive chondrodysplasia punctata could be observed. His mother had lowered steroid sulphatase activity compatible with carriership for X-linked ichthyosis and a submicroscopic translocation (X;14)(p22.31;p11.1). This finding combined with a normal amplification of exons 1, 5, and 10 of the STS gene from propositus' DNA suggested a breakpoint upstream of the STS gene. The submicroscopic maternal translocation had important implications for genetic counseling. This case report illustrates that contiguous gene syndrome related to the Xpter region may have an atypical clinical presentation and the usefulness of combined clinical, biochemical, molecular, and fluorescence in situ hybridization analysis.
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http://dx.doi.org/10.1002/(sici)1096-8628(19991119)87:2<189::aid-ajmg12>3.3.co;2-h | DOI Listing |
J Clin Endocrinol Metab
January 2025
Division of Orthogenetics, Department of Pediatrics, Nemours Children's Hospital, Delaware, 1600 Rockland Road, Wilmington, DE, 19803, USA.
Achondroplasia is the most common disproportionate short-stature skeletal dysplasia. Features associated with achondroplasia are rhizomelia, macrocephaly, midface hypoplasia, and typical cognition. Potential medical complications include foramen magnum stenosis, hydrocephalus, middle ear dysfunction, obstructive and central sleep apnea, spinal stenosis and genu varum.
View Article and Find Full Text PDFPatient Relat Outcome Meas
January 2025
Novo Nordisk, Søborg, 2860, Denmark.
Purpose: Growth hormone deficiency (GHD) causes decreased growth rate in children, resulting in short stature in childhood and adulthood. Daily subcutaneous injections with growth hormone (GH) have been standard treatment. Newer weekly GH formulations now exist.
View Article and Find Full Text PDFJPRAS Open
March 2025
Department of Plastic and Reconstructive Surgery, University of the Ryukyu Hospital, Okinawa, Japan.
Total pharyngo-laryngo-esophagectomy (TPLE) with free jejunal transplantation (FJT) is the standard reconstructive procedure for hypopharyngeal cancer, typically utilizing the superior thyroid artery as the recipient vessel. However, patient-specific anatomical variations and comorbidities can significantly complicate this surgery. We present a unique case of a 68-year-old male with hypopharyngeal cancer who exhibited multiple challenges, including short stature (126 cm), low weight (35 kg), cervical spondylosis, and a history of vertebroplasty, highlighting the complexities inherent in such reconstructions.
View Article and Find Full Text PDFAnn Pediatr Endocrinol Metab
January 2025
Department of Medicine, Surgery and Health Science, University of Trieste, Trieste, Italy.
Long-acting growth hormones (LAGHs) represent a significant advancement in the treatment of pediatric growth hormone deficiency (GHD), offering an alternative to daily recombinant human growth hormone (rhGH) therapy. Traditional rhGH treatments, while effective, require daily injections, often leading to poor adherence due to the frequency of dosing, injection pain, and difficulties with storage and travel. In contrast, LAGHs, such as somatrogon, somapacitan, and lonapegsomatropin, are designed for once-weekly administration, improving patient compliance and quality of life.
View Article and Find Full Text PDFMed Sci Sports Exerc
November 2024
Department of Biomedical Sciences for Health, Università degli Studi di Milano, ITALY.
Purpose: Cigarette smoking (CS) induces systemic changes that impair cardiorespiratory and muscular function both at rest and during exercise. Although these abnormalities are reported in sedentary, middle-aged smokers (SM) with pulmonary disease, few and controversial studies focused on young, physically active SM at the early stage of smoking history. This study aimed at assessing the impact CS on cardiorespiratory and metabolic response during an incremental test and the subsequent recovery in young, physically active SM without known lung or cardiovascular disease.
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