Beta thalassemia is an inherited blood disorder that results in inefficient erythropoiesis due to genetic mutation that leads to the reduction or absence of the hemoglobin beta-globulin protein. Approximately 8.5% of UAE residents suffer from β-thalassemia, a significant health and financial problem. The treatment options available for β-Thalassemia major are limited and associated with a wide range of complications. β-thalassemia gene therapy is emerging as a potential novel treatment option that eliminates the complications caused by the current long-term treatment modalities and the associated economic burden. This paper reviews the scientific literature related to emerging gene therapy for β-Thalassemia by analyzing all the articles published from January 2010 to December 2023 in the English language on Databases like PubMed, Scopus, ProQuest, and CINAHL. The use of gene therapy has demonstrated promising outcomes for a permanent cure of β-Thalassemia. To conclude, gene therapy is an innovative solution. It demonstrates a promising future, but does come with its own setbacks and is something that must be tackled in order to revolutionize it in the medical world. FDA-approved ZYNTEGLO is a potentially one-time curative treatment for β-Thalassemia. Although cutting-edge, its use is limited because of the high cost—a price of USD 2.8 million per patient.

Attention deficit hyperactivity disorder (ADHD), once considered primarily a childhood condition, is now increasingly recognized as a disorder that persists into adulthood and significantly impacts academic and professional success. The aim of this study was to investigate the prevalence of ADHD and identify associated risk factors among university students. A cross-sectional study was conducted over a period of six months at Ras Al Khaimah Medical and Health Sciences University, United Arab Emirates. Data were collected through a self-report questionnaire addressing sociodemographic, lifestyle, and family environment factors. ADHD symptoms were assessed using the World Health Organization Adult ADHD Self-Report Scale version 1.1, based on the Diagnostic and Statistical Manual of Mental Disorders - Fifth Edition criteria. The study sample included 472 students, with a mean age of 19.9±1.85 years. The overall prevalence of ADHD was 13.6% (n=64). Logistic regression analysis revealed significant associations between the mother's smoking status (odds ratio (OR): 2.35; 95% confidence interval (95%CI): 1.949–5.862, p=0.050) and living in shared housing (OR: 3.35; 95%CI: 1.674–6.723, p=0.001) with increased odds of ADHD. Conversely, being male (OR: 0.4; 95%CI: 0.216–0.891, p=0.02) and being born full-term (OR: 0.331; 95%CI: 0.138–0.794, p=0.013) were associated with decreased odds of ADHD. Other factors, such as college affiliation, smoking status, exercise habits, maternal employment, mode of delivery, and pregnancy complications, were not significant risk factors. These findings highlight the need for targeted interventions in university settings, including early screening, tailored support services, and increased staff awareness, to support students with ADHD and enhance their academic success and well-being.

Beta thalassemia is an inherited blood disorder that results in inefficient erythropoiesis due to genetic mutation that leads to reduction or absence of the hemoglobin beta-globulin protein. Approximately 8.5% of UAE residents suffer from β-thalassemia, a significant health and financial problem. The treatment options available for β-Thalassemia major are limited and associated with a wide range of complications. β-thalassemia gene therapy is emerging as a potential novel treatment option that eliminates the complications caused by the current long-term treatment modalities and the associated economic burden. This paper reviews the scientific literature related to emerging gene-therapy for β-Thalassemia by analyzing all the articles published from January 2010 to December 2023 in the English language on Databases like PubMed, Scopus, ProQuest, and CINAHL. The use of gene therapy has demonstrated promising outcomes for a permanent cure of β-Thalassemia. ZYNTEGLO (Betibeglogene autotemcel) is the only cell-based gene therapy which was approved by the FDA on 19th August, 2018. To conclude, Gene therapy is an innovative solution. It demonstrates a promising future, but does come with its own set-backs and is something that must be tackled in-order to revolutionize it in the medical world. FDA approved ZYNTEGLO is a potentially one-time curative treatment for β-Thalassemia. Although cutting-edge, its use is limited because of the high cost; a price of $2.8million per patient.

West syndrome is a distinctive type of epilepsy characterized by infantile spasms, hypsarrhythmia on EEG, and developmental regression. It affects children between 4 months and 2 years old, with an incidence of 2–3.5 per 10,000 births, being more common in boys. The syndrome is classified into symptomatic, idiopathic, and cryptogenic forms, based on underlying causes such as brain trauma, malformations, infections, chromosomal abnormalities (e.g., Tuberous Sclerosis Complex), or genetic mutations in genes like ARX and CDKL5. This article presents a case of West syndrome in an 8‐months‐old female without a known genetic cause, despite extensive genetic analysis. The patient presented to us with unprovoked spasms occurring over 2 months. Evaluation through EEG confirmed modified hypsarrhythmia, and MRI revealed significant brain lesions. A comprehensive metabolic panel was negative, and whole exome sequencing for the patient and her parents was inconclusive. This finding is not uncommon, as an estimated 40% of West syndrome cases remain without a confirmed genetic diagnosis. Current treatment recommendations by pediatric neurologists in the GCC include first‐line therapy with vigabatrin alone or in combination with high‐dose oral corticosteroids. If no response occurs within 2 weeks, ACTH or corticosteroids are added. ACTH, though effective, is less favored due to accessibility, cost, and relapse rates. Vigabatrin has the fewest side effects, followed by corticosteroids, with ACTH presenting the most, particularly irritability and hypertension. This case highlights the challenges in diagnosing and managing West syndrome, especially with inconclusive genetic analysis. It emphasizes the need for continued advancements in genetic diagnostics to uncover new mutations and improve patient outcomes.