Pathogenic variants in DDX3X are associated with neurodevelopmental disorders. Communication impairments are commonly reported, yet specific speech and language diagnoses have not been delineated, preventing prognostic counseling and targeted therapies. Here, we characterized speech and language in 38 female individuals, aged 1.69-24.34 years, with pathogenic and likely pathogenic DDX3X variants (missense, n = 13; nonsense, n = 12; frameshift, n = 7; splice site, n = 3; synonymous, n = 2; deletion, n = 1). Standardized speech, language, motor, social, and adaptive behavior assessments were administered. All participants had gross motor deficits in infancy (34/34), and fine motor deficits were common throughout childhood (94%; 32/34). Intellectual disability was reported in 86% (24/28) of participants over 4 years of age. Expressive, receptive, and social communication skills were, on average, severely impaired. However, receptive language was significantly stronger than expressive language ability. Over half of the assessed participants were minimally verbal (66%; 22/33; range = 2 years 2 months-24 years 4 months; mean = 8 years; SD = 6 years) and augmented speech with sign language, gestures, or digital devices. A quarter of the cohort had childhood apraxia of speech (25%; 9/36). Despite speech and language impairments, social motivation was a relevant strength. Many participants used augmentative and alternative communication (AAC), underscoring the need for early, tailored, and comprehensive AAC intervention.
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http://dx.doi.org/10.1002/ajmg.b.32971 | DOI Listing |
Genet Med
December 2024
Sheffield Clinical Genetics Service, Sheffield Children's NHS Foundation Trust, Sheffield, UK; Division of Clinical Medicine, University of Sheffield, Sheffield, UK. Electronic address:
Purpose: The TAOK proteins are a group of serine/threonine-protein kinases involved in signalling pathways, cytoskeleton regulation, and neuronal development. TAOK1 variants are associated with a neurodevelopmental disorder (NDD) characterized by distinctive facial features, hypotonia and feeding difficulties. TAOK2 variants have been reported to be associated with autism and early-onset obesity.
View Article and Find Full Text PDFArch Phys Med Rehabil
December 2024
Spinal Cord Injury Center, BG Trauma Center Murnau, Murnau, Germany; ParaMove, SCI Research Unit, BG Trauma Center Murnau, Murnau, Germany; Institute of Molecular Regenerative Medicine, Paracelsus Medical University, Salzburg, Austria. Electronic address:
Objective: The pathophysiological mechanisms of dysphagia in individuals with traumatic cervical spinal cord injury (SCI) are not well understood yet. Several risk factors for developing dysphagia after SCI were postulated including mechanical ventilation, tracheostomy, age, female sex, anterior surgical approach, SCI severity, and multi-level spinal fusion. This study aimed to identify risk factors for dysphagia in individuals who sustained traumatic cervical SCI.
View Article and Find Full Text PDFInt J Lang Commun Disord
December 2024
Department of Clinical Speech and Language Studies, Trinity College Dublin, Dublin, Ireland.
Background: Individuals with dementia have communication limitations resulting from cognitive impairments that define the syndrome. Whereas there are numerous cognitive assessments for individuals with dementia, there are far fewer communication assessments. The Profiling Communication Ability in Dementia (P-CAD) was developed to address this gap.
View Article and Find Full Text PDFJAMIA Open
February 2025
Hasso Plattner Institute for Digital Engineering, University of Potsdam, Potsdam 14482, Germany.
Objective: To improve performance of medical entity normalization across many languages, especially when fewer language resources are available compared to English.
Materials And Methods: We propose xMEN, a modular system for cross-lingual (x) medical entity normalization (MEN), accommodating both low- and high-resource scenarios. To account for the scarcity of aliases for many target languages and terminologies, we leverage multilingual aliases via cross-lingual candidate generation.
World J Gastroenterol
December 2024
School of Medicine, National Yang Ming Chiao Tung University, Taipei 112, Taiwan.
Background: Diagnosing laryngopharyngeal reflux (LPR) is challenging due to overlapping symptoms. While proton pump inhibitors (PPIs) are commonly prescribed, reliable predictors of their responsiveness are unclear. Reflux monitoring technologies like dual potential of hydrogen (pH) sensors and multichannel intraluminal impedance-pH (MII-pH) could improve diagnosis.
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