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Current and Emerging Therapies for Lysosomal Storage Disorders.
Drugs
January 2025
Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust, University College London, London, NW3 2QG, UK.
Lysosomal storage disorders (LSDs) are rare inherited metabolic disorders characterized by defects in the function of specific enzymes responsible for breaking down substrates within cellular organelles (lysosomes) essential for the processing of macromolecules. Undigested substrate accumulates within lysosomes, leading to cellular dysfunction, tissue damage, and clinical manifestations. Clinical features vary depending on the degree and type of enzyme deficiency, the type and extent of substrate accumulated, and the tissues affected.
View Article and Find Full Text PDFPharmacologic Management of Heart Failure with Preserved Ejection Fraction (HFpEF) in Older Adults.
Drugs Aging
January 2025
Program for the Care and Study of the Aging Heart, Department of Medicine, Weill Cornell Medicine, 420 East 70th St, New York, NY, LH-36510063, USA.
There are several pharmacologic agents that have been touted as guideline-directed medical therapy for heart failure with preserved ejection fraction (HFpEF). However, it is important to recognize that older adults with HFpEF also contend with an increased risk for adverse effects from medications due to age-related changes in pharmacokinetics and pharmacodynamics of medications, as well as the concurrence of geriatric conditions such as polypharmacy and frailty. With this review, we discuss the underlying evidence for the benefits of various treatments in HFpEF and incorporate key considerations for older adults, a subpopulation that may be at higher risk for adverse drug events.
View Article and Find Full Text PDFFecal metabolomic analysis of the role of gut microbiota and short-chain fatty acids in the therapeutic mechanism of Timosaponin AIII in Sjögren's syndrome.
Clin Rheumatol
January 2025
Department of Rheumatology, Guang'anmen Hospital, Chinese Academy of Chinese Medical Sciences, Beijing, People's Republic of China.
Introduction/objectives: Sjogren's syndrome (SS) is a chronic inflammatory and difficult-to-treat autoimmune disease. Timosaponin AIII (TAIII), a plant-derived steroidal saponin, effectively inhibits cell proliferation, induces apoptosis, and exhibits anti-inflammatory properties. This study explored the mechanisms of action of TAIII in SS treatment by studying gut microbiota and short-chain fatty acids (SCFAs) using fecal metabolomics.
View Article and Find Full Text PDFClinical and laboratory characteristics of Sjögren's syndrome-associated autoimmune liver disease: a real-world, 10-year retrospective study.
Clin Rheumatol
January 2025
Department of Rheumatology and Immunology, Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology, Wuhan, China.
Objectives: To investigate the clinical and laboratory features of Sjögren's syndrome-associated autoimmune liver disease (SS-ALD) patients and identify potential risk and prognostic factors.
Methods: SS patients with or without ALD, who visited Tongji Hospital between the years 2011 and 2021 and met the 2012 American College of Rheumatology (ACR) classification criteria for Sjögren's syndrome, were retrospectively enrolled. The clinical and laboratory data of the enrolled patients, including autoimmune antibodies, were collected and analyzed with principal component analysis, correlation analysis, LASSO regression, and Cox regression.
Pancreas-guided C-shaped surgical procedure: a safer and more efficient procedure for laparoscopic left hemicolectomy in obese patients.
Updates Surg
January 2025
Department of Gastrointestinal Surgery, The First People's Hospital of Foshan, No. 81 Lingnan Avenue North, Foshan, China.
The surgical risk is higher for obese patients undergoing laparoscopic left hemicolectomy. To enhance the surgical safety and efficacy for obese patients, we have innovatively integrated the advantages of various surgical approaches to modify a pancreas-guided C-shaped surgical procedure. The safety and quality were assessed through a retrospective analysis.
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