Hereditary angioedema (HAE) is a rare autosomal dominant disorder marked by episodic, non-urticarial swelling due to C1 esterase inhibitor (C1-INH) deficiency or dysfunction, leading to excessive bradykinin-mediated vascular permeability. While current treatments focus on symptomatic control using C1-INH replacement or kallikrein inhibitors, they require frequent administration and do not address the underlying genetic defect. CRISPR-Cas9 gene-editing technologies, particularly the investigational therapy NTLA-2002, offer a transformative approach by targeting the KLKB1 gene to durably reduce plasma kallikrein levels. This narrative review summarizes the pathophysiology and conventional treatments of HAE, and highlights emerging clinical evidence supporting the safety and efficacy of NTLA-2002. Preliminary trials demonstrate substantial reductions in HAE attack frequency and plasma kallikrein, with minimal adverse events. However, concerns about long-term safety, off-target effects, ethical implications, and accessibility remain. CRISPR-based therapeutics such as NTLA-2002 represent a paradigm shift in the management of HAE and underscore the broader potential of in vivo gene editing for genetic disorders.

Background: Obesity has become a major global health concern and has been frequently associated with numerous pathological conditions, one of which is kidney stones, also known as nephrolithiasis. Both these conditions present a significant burden on healthcare systems, and their coexistence amplifies the challenges for both the patients as well as the healthcare systems. To develop effective management strategies, it is crucial to understand the pathophysiological mechanisms connecting these two conditions. Aim: This study aims to explore the pathophysiological mechanisms linking obesity and kidney stone formation, along with presenting cost-effective strategies, including dietary and lifestyle modifications, which can help decrease the incidence of obesity-related kidney stone formation. Methodology: This study is performed by reviewing reporting items, including systematic reviews and meta-analyses, using electronic databases such as PubMed and Google Scholar. The search term for this purpose was “Obesity AND Kidney Stones,” and articles were reviewed from 2002 to 2024. Result: The study displays that obesity increases the risk of kidney stone formation through multiple pathways. Increased uric acid excretion, increased calcium levels, insulin resistance, and a sedentary way of living contribute to the pathophysiology of kidney stones. The study also analyzes the economic burden of these diseases and explores cost-effective strategies for their management. Conclusion: The risk of kidney stones in obese people can be reduced by using individualized strategies. Improving daily physical activity levels and healthy diet intake has proved to be increasingly important in the prevention of obesity-related kidney stones.

Background and Aims: Cerebrotendinous xanthomatosis (CTX) is a rare autosomal recessive disorder caused by mutations in the CYP27A1 gene, leading to deficient sterol 27‐hydroxylase activity. This enzyme is critical for bile acid synthesis, and its dysfunction results in reduced chenodeoxycholic acid (CDCA) levels and subsequent accumulation of cholestanol in tissues. This study highlights the recent FDA approval of Ctexli (chenodiol), a synthetic CDCA formulation, as the first treatment option for CTX. Methods: A comprehensive literature search was performed using PubMed and Google Scholar, and relevant articles were identified that focused on the mechanism of action, clinical efficacy, and safety of chenodiol. Results: Data from the RESTORE Phase 3 trial demonstrated that chenodiol significantly reduced plasma cholestanol and urinary bile alcohol levels, with the most pronounced results observed in patients treated before the onset of irreversible neurological damage. The FDA approval of Ctexli validated these findings and established chenodiol as a reliable therapeutic option for CTX. Conclusion: The FDA approval of chenodiol marks a significant milestone in the management of CTX, providing a standardized and evidence‐based therapy for a previously neglected condition. Early diagnosis, broader screening, and global access to the drug will be key to improving outcomes and ensuring lasting clinical benefit.

Objective: While several meta-analyses have evaluated the cardiovascular (CV) and renal benefits of GLP-1 receptor agonists, including semaglutide, none have specifically investigated its impact on stroke risk reduction. This meta-analysis aims to evaluate the overall effect of semaglutide on reducing stroke risk among populations at high CV-risk. Methods: A comprehensive search was undertaken across PubMed, Scopus, and Cochrane databases up to May 2024. Four eligible studies included randomized controlled trials (RCTs) that compared semaglutide with placebo for cardiovascular outcomes in both diabetic and non-diabetic populations. Primary cerebrovascular endpoints included stroke and its subtypes. Secondary cardiovascular endpoints were all-cause mortality, major adverse cardiovascular events (MACE), and CV-mortality. Statistical analysis was conducted using Review Manager 5.4 and the risk ratios (RR) with 95% confidence interval (CI) were computed employing a random-effects model. Results: Semaglutide demonstrated a modest reduction in stroke risk (RR 0.84, 95% CI 0.70–1.00, P=0.050, I²=21%), approaching statistical significance. It also demonstrated significant benefits in reducing all-cause mortality (RR 0.81, 95% CI 0.70-0.94, P=0.006, I2=42%), MACE (RR 0.81, 95% CI 0.74-0.88, P<0.001, I2=0%), and cardiovascular mortality (RR 0.79, 95% CI 0.67-0.93, P=0.006, I2=28%). Conclusion: The secondary outcomes were significantly reduced, stroke risk reduction was marginal, highlighting semaglutide’s promising vascular benefits. The inclusion of only four RCTs and moderate heterogeneity among the trials may limit the generalizability of these findings.

Background and Aims: Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare acquired clonal blood disorder caused by mutations in the PIGA gene, leading to complement‐mediated hemolysis. Currently available terminal complement inhibitors, such as Eculizumab and Ravulizumab, pose several challenges, including the need for frequent intravenous infusions and the potential for resistance due to C5 polymorphisms. This study highlights the clinical significance of Crovalimab, a novel C5 inhibitor developed using SMART‐antibody technology, as a promising alternative. Methods: An extensive literature review was conducted using PubMed to evaluate the pharmacological properties, mechanism of action, and clinical trial data of Crovalimab. Phase 3 trials—COMMODORE 1, 2, and 3—were analyzed to assess Crovalimab's safety, efficacy, and potential benefits in both C5‐inhibitor naïve and previously treated patients. Results: Crovalimab demonstrated high bioavailability, an extended half‐life, and subcutaneous administration every 4 weeks, offering a better alternative to intravenous therapies. Unlike existing treatments, Crovalimab targets the C5 β‐chain, making it effective even in patients with the R885H polymorphism. The COMMODORE trials reported favorable outcomes, including effective hemolysis control, reduced transfusion dependence, and a manageable safety profile. Adverse events were mostly mild, with rare occurrences of transient immune complex reactions. Conclusion: Crovalimab represents a significant advancement in the management of PNH, with the potential to reduce treatment burden while maintaining efficacy. However, further research is required to evaluate its long‐term safety and effectiveness across diverse populations.