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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 PDFRecent Advances in Radical Coupling Reactions Directly Involving Bicyclo[1.1.1]pentane (BCP).
Top Curr Chem (Cham)
January 2025
School of Pharmacy, Hangzhou Normal University, Hangzhou, 311121, Zhejiang, People's Republic of China.
BCP (bicyclo[1.1.1]pentane) is an ideal saturated carbon bioisostere, instead of the traditional benzene group, which has been extensively developed.
View Article and Find Full Text PDFAwareness of Contraindications Among Cancer Patients in Riyadh, Saudi Arabia: A Cross-Sectional Study.
Cancer Control
January 2025
Department of Pharmacy Practice, College of Pharmacy, AlMaarefa University, Dariyah, Riyadh, Saudi Arabia.
Introduction: Cancer patients often face challenges in managing their disease, particularly with regard to contraindications related to medications, foods, and physical activity, which can negatively affect treatment outcomes. This study aimed to evaluate cancer patients' awareness of these contraindications and to explore the influence of sociodemographic factors, support systems, comorbidities, and medication use on their knowledge.
Methods: A cross-sectional prospective study was conducted with 125 cancer patients in Saudi Arabia between December 2022 and February 2023.
[Assessment of corticosteroid use as a marker of quality-of-care in the management of inflammatory bowel disease].
Rev Gastroenterol Peru
January 2025
Servicio de Gastroenterología, Hospital Clínico de la Universidad de Chile, Universidad de Chile, Santiago, Chile.
Introduction: Despite advancements in therapeutic strategies, corticosteroids continue to play a role in inducing remission in Inflammatory Bowel Disease (IBD). Unfortunately, these drugs are often misused.
Objectives: To assess the dose and duration of corticosteroid therapy,and the subsequent change in treatment among patients with IBD.
Sotatercept in pulmonary hypertension and beyond.
Eur J Clin Invest
January 2025
Department of Surgical, Medical and Molecular Pathology and Critical Area, Laboratory of Biochemistry, University of Pisa, Pisa, Italy.
Sotatercept binds free activins by mimicking the extracellular domain of the activin receptor type IIA (ACTRIIA). Additional ligands are BMP/TGF-beta, GDF8, GDF11 and BMP10. The binding with activins leads to the inhibition of the signalling pathway and the deactivation of the bone morphogenic protein (BMP) receptor type 2.
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