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Introduction/Aims: New therapeutic strategies to increase survival motor neuron protein levels in spinal muscular atrophy (SMA) have focused on replacing the SMN1 gene. Onasemnogene abeparvovec was approved by the US Food and Drug Administration in 2019 for treatment of children with SMA less than 2 years of age. Postmarketing studies are limited, especially outside of Europe and the United States. Herein we describe a single‐center experience with onasemnogene abeparvovec from the Middle East. Methods: Between November 17, 2020 and January 31, 2022, 25 children with SMA received onasemnogene abeparvovec at our center in the United Arab Emirates. Data were collected on patients' demographics, age at diagnosis, SMA type, genetic information, relevant medical history, laboratory investigations, and Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP‐INTEND) functional assessment scores at baseline and 1 and 3 months after gene therapy. Results: Onasemnogene abeparvovec was well tolerated. Significant improvements in CHOP‐INTEND scores were observed after the therapy. Elevation of liver enzymes and thrombocytopenia were the most common adverse events, but were transient and managed with high‐dose corticosteroids. There were no life‐threatening adverse events or deaths reported during the 3‐month follow‐up period. Discussion: The study findings concurred with those of previously published studies. Side effects of gene transfer therapy are well tolerated, although serious complications can arise. In such cases, persistent transaminitis for example, steroid dose escalation is warranted with close observation of the patient's clinical status and lab values. Combination therapy should be explored as an alternative to gene transfer therapy only. [ABSTRACT FROM AUTHOR]

This paper reviews the state-of-the-art in sensing technologies that are relevant for Autism Spectrum Disorder (ASD) screening and therapy. This disorder is characterized by difficulties in social communication, social interactions, and repetitive behaviors. It is diagnosed during the first three years of life. Early and intensive interventions have been shown to improve the developmental trajectory of the affected children. The earlier the diagnosis, the sooner the intervention therapy can begin, thus, making early diagnosis an important research goal. Technological innovations have tremendous potential to assist with early diagnosis and improve intervention programs. The need for careful and methodological evaluation of such emerging technologies becomes important in order to assist not only the therapists and clinicians in their selection of suitable tools, but to also guide the developers of the technologies in improving hardware and software. In this paper, we survey the literatures on sensing technologies for ASD and we categorize them into eye trackers, movement trackers, electrodermal activity monitors, tactile sensors, vocal prosody and speech detectors, and sleep quality assessment devices. We assess their effectiveness and study their limitations. We also examine the challenges faced by this growing field that need to be addressed before these technologies can perform up to their theoretical potential. [ABSTRACT FROM AUTHOR]

Stroke is often viewed as a diagnosis found In the elderly with or without comorbidities, but it is vital to not rule it out in a pediatric patient presenting with signs and symptoms of stroke. Here, we present a case of an 18-month-old boy who arrived at the emergency department with left arm weakness and left-sided seizures a few minutes after a right-sided trivial neck bite that was initially overlooked by the parents until symptoms occurred. Urgent imaging further with a computed tomography scan of the brain revealed a hypodense lesion in the area covering the lateral part of the frontal lobe, insula, and parietal cortex of the right hemisphere. Subsequent CT cervical-cerebral angiogram revealed normal aortic arch, carotid, and vertebral arteries with no dissection, stenosis, or occlusion. However, there was a 4 mm-long occlusion of the M2 segment of the right middle cerebral artery (MCA) suggestive of emboli and subsequent low attenuation of the brain parenchyma in the anterior aspect of the right MCA vascular territory corresponding with the infarction. The objective of this case report is to educate and inform both parents and medical professionals regarding the risk of neurological damage that can occur with minor head and neck trauma that is often overlooked and therefore the importance of ruling it out with necessary imaging modalities., Competing Interests: The authors have declared that no competing interests exist., (Copyright © 2024, Duvuru et al.)