Recently, an autosomal recessive disorder including the triad of microcephaly, infantile epileptic encephalopathy, and permanent neonatal diabetes syndrome (MEDS, OMIM#614231) has emerged as a new distinguishing syndrome. Eight cases of whom seven from Arab countries, have been reported in association with biallelic variants in the IER3IP1 gene (Immediate early response-3 interacting protein-1). Here, we describe a Tunisian boy who presented with permanent neonatal diabetes, microcephaly, generalized seizures and hypovirilized external genitalia consisting of a small genitalia and unilateral cryptorchidism. Chromosomal analysis indicated a 46, XY karyotype in all metaphases. Exome sequencing identified a homozygous missense variant (c.62 T > G; p. Val21Gly) in the IER3IP1 gene, that is predicted to alter the protein structure within the hydrophobic/transmembrane. This variant was previously reported in two cases associated with MEDS. This is the first reported case of MEDS in Tunisia. Our report focuses on the IER3IP1 related phenotypic spectrum and assumes abnormal genitalia as part of the syndrome. Consequently, we recommend to perform hormonal testing on this topic to understand the effect of the IER3IP1 variant on the male genital pathway.
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http://dx.doi.org/10.1016/j.ejmg.2021.104285 | DOI Listing |
J Clin Med
December 2024
Unit of Hand Surgery, Microsurgery and Reconstructive, Department of Orthopaedics and Traumatology, CTO Hospital, 10126 Turin, Italy.
Neonatal brachial plexus palsy (NBPP) is a flaccid paralysis of the upper limbs that occurs in about 0.4 percent of live births. This condition can produce permanent disabilities; to date, there is no consensus on protocols to be applied for the rehabilitation of children with this condition.
View Article and Find Full Text PDFJ Neurosurg Case Lessons
January 2025
Department of Neurosurgery, Medical University of South Carolina, Charleston, South Carolina.
Background: Myelomeningocele and sagittal craniosynostosis are 2 neurosurgical pathologies with complications such as increased intracranial pressure (ICP) and hydrocephalus. While the 2 defects commonly occur independently, their simultaneous occurrence is exceptionally rare.
Observations: The authors report the case of a newborn male diagnosed with a simultaneous myelomeningocele and sagittal craniosynostosis.
Neoreviews
January 2025
Division of Neonatology, Department of Pediatrics, University of Maryland School of Medicine, Baltimore, Maryland.
Advances in neonatal medicine over the last several decades have led to improvements in survival for the most fragile and critically ill infants. A mainstay of supporting the health needs of hospitalized infants involves use of peripheral intravenous (PIV) catheters to administer medications, parenteral nutrition, and blood products. However, PIV catheters are not without risks and complications such as infiltration and extravasation.
View Article and Find Full Text PDFMed J Armed Forces India
December 2024
Director & Commandant, Armed Forces Medical College, Pune, India.
Neonatal diabetes mellitus is a rare disorder with prevalence of one in 400,000 live births that's defined by persistent hyperglycaemia within the first six months of life. Neonatal diabetes is heterogeneous and can be transient or permanent. Developmental delay, Epilepsy and Neonatal Diabetes (DEND) syndrome is characterised by developmental delay, epilepsy, and neonatal diabetes.
View Article and Find Full Text PDFJCEM Case Rep
January 2025
Department of Paediatric Endocrinology, Kandang Kerbau Women's and Children's Hospital, 229899 Singapore.
Maturity-onset diabetes of the young (MODY) represents 1% to 5% of patients with diabetes mellitus (DM), and numerous genes associated with MODY have been identified. While mutations of the insulin gene () are known to cause permanent neonatal DM, rare disease-causing variants have also been found in MODY. These patients demonstrate variable clinical phenotypes-from milder forms requiring lifestyle or oral agent interventions to severe forms requiring lifelong insulin.
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