Fazirsiran for the treatment of alpha-1 antitrypsin deficiency-associated liver disease findings from the SEQUOIA phase 2 trial.

Alpha-1 antitrypsin deficiency (AATD) is a prevalent autosomal recessive disorder affecting approximately 3 million people worldwide, caused by mutations in the SERPINA1 gene leading to low alpha-1 antitrypsin (AAT) levels. This deficiency predisposes individuals to chronic obstructive pulmonary disease, bronchiectasis, neonatal cholestasis, and liver cirrhosis. The most common pathogenic mutation, PI*ZZ allele, produces misfolded Z-AAT protein accumulating in hepatocytes.

Colchicine as an Adjunct Therapy Postablation for Atrial Fibrillation: A Systematic Review and Meta-analysis.

Atrial fibrillation (AF) recurrence following catheter ablation remains a significant clinical challenge, often attributed to postprocedural inflammation. Colchicine, an anti-inflammatory agent, has emerged as a potential therapeutic strategy to reduce recurrence. This meta-analysis evaluates the efficacy and safety of colchicine in preventing AF recurrence after ablation.

Beyond blood transfusions: exploring iron chelation therapies in transfusion-dependent beta-thalassemia.

Introduction: Abnormal hemoglobin, or hemoglobinopathy, affects about 7% of the global population. Major hemoglobinopathies like beta-thalassemia and sickle cell disease require regular blood transfusions, leading to chronic iron overload. This review examines the efficacy and safety of deferiprone, an oral iron chelator, in managing iron overload in pediatric patients with transfusion-dependent conditions.

Cavernous Hemangioma of the Palatine Tonsil: A Case Report and Review of the Literature.

Unlabelled: Cavernous hemangiomas are venous malformations characterized by interconnected channels of abnormally dilated blood vessels having low flow of blood. Whilst venous malformations most commonly affect the head and neck region, isolated cavernous hemangiomas of the palatine tonsils are extremely rare; with only 4 cases being reported in the English Literature. We report a case of a 43-year-old female who was referred to us for the evaluation and management of a right tonsillar swelling.

Efficacy and safety of the FGF19 analog aldafermin for the treatment of nonalcoholic steatohepatitis: a GRADE assessed systematic review and meta-analysis.

Background: Nonalcoholic fatty liver disease (NAFLD) is increasingly concerning due to its rising prevalence. It encompasses conditions from simple steatosis to severe nonalcoholic steatohepatitis (NASH), posing risks such as fibrosis, cirrhosis, or hepatocellular carcinoma if untreated. This systematic review and meta-analysis aims to assess aldafermin, an FGF19 analog, for efficacy and safety in NASH patients.

Efficacy and safety of resmetirom for the treatment of nonalcoholic steatohepatitis: a GRADE assessed systematic review and meta-analysis.

There are no Food and Drug Administration (FDA)-approved treatment options for nonalcoholic steatohepatitis (NASH) which is a prevailing disease that leads to fibrosis, cirrhosis, or hepatocellular carcinoma. Hence, this systematic review and meta-analysis aims to determine the efficacy and safety of resmetirom, the first FDA-approved drug, for the treatment of NASH. A Grading of Recommendations, Assessment, Development, and Evaluation assessed systematic search of Cochrane Library , MEDLINE , Scopus , and Google Scholar database was conducted from inception till 31 March 2024.

Evaluation of non-invasive diagnostic tools for diarrhea: a systematic review of point-of-care tests and biomarkers.

Background: Diarrhea is a prevalent condition affecting millions worldwide. However, current standard diagnostic methods have many drawbacks. This review examines various non-invasive point-of-care (POC) tests and biomarkers aiding rapid diagnosis of diarrhea from different causes.