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Pharmacological Aspects in the Management of Children and Adolescents with Prader-Willi Syndrome.
Paediatr Drugs
January 2025
Division of Endocrinology, Department of Pediatrics, University of Florida, PO Box 100296, Gainesville, FL, 32610, USA.
Prader-Willi syndrome is a rare neurodevelopmental disorder that impacts the musculoskeletal, endocrine, pulmonary, neurologic, ocular, and gastrointestinal systems. In addition, individuals with Prader-Willi syndrome have issues with cognitive development, characteristic behavioral problems, and perhaps most profoundly, appetite control. Currently, the only US Food and Drug Administration-approved therapy for Prader-Willi syndrome is growth hormone, which has been Food and Drug Administration approved for > 20 years for the treatment of growth failure in Prader-Willi syndrome.
View Article and Find Full Text PDFInavolisib: First Approval.
Drugs
January 2025
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Inavolisib (Itovebi) is an orally administered, phosphatidylinositol-3-kinase alpha (PI3Kα) inhibitor being developed by Genentech, a member of the Roche group, for the treatment of solid tumours. On 10 October 2024, inavolisib received its first approval in the USA in combination with palbociclib and fulvestrant for the treatment of adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth factor 2 (HER2)-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy. In the EU and other countries worldwide, regulatory review of inavolisib is currently underway.
View Article and Find Full Text PDFPerioperative chemotherapy strategies in diffuse gastric cancer.
World J Gastrointest Surg
January 2025
Department of Surgery, Division of Endocrine & Minimally Invasive Surgery, Weill Cornell Medical College, New York-Presbyterian Hospital, New York, NY 10128, United States.
This study reviews the findings of a recent study by Li , which demonstrated that perioperative chemotherapy benefits patients with diffuse-type gastric cancer compared to surgery alone. Despite potential biases, the study supports the inclusion of perioperative chemotherapy in treatment guidelines. Neoadjuvant and adjuvant chemotherapy may also provide similar survival outcomes, allowing for flexible treatment planning.
View Article and Find Full Text PDFIndocyanine green fluorescence-guided laparoscopic central pancreatectomy for complete pancreatic transection trauma: a rare case and literature review.
Front Surg
January 2025
Department of Hepato-Pancreato-Biliary Surgery, Hunan Provincial People's Hospital (The First Affiliated Hospital of Hunan Normal University), Changsha, Hunan, China.
Background: Pancreatic trauma is a rare solid organ injury. Conservative treatment is often indicated in patients with no pancreatic duct injury, while patients with high-grade pancreatic damage most often require surgical intervention. Laparoscopic central pancreatectomy (LCP) is a parenchyma-sparing approach and can prevent endocrine and exocrine insufficiency after pancreatic resection.
View Article and Find Full Text PDFIdentification of a pancreatic stellate cell gene signature and lncRNA interactions associated with type 2 diabetes progression.
Front Endocrinol (Lausanne)
January 2025
Huangjiang Hospital, Dongguan, Guangdong, China.
Background: Type 2 diabetes (T2D) has become a significant global health threat, yet its precise causes and mechanisms remain unclear. This study aims to identify gene expression patterns specific to T2D pancreatic islet cells and to explore the potential role of pancreatic stellate cells (PSCs) in T2D progression through regulatory networks involving lncRNA-mRNA interactions.
Methods: In this study, we screened for upregulated genes in T2D pancreatic islet samples using bulk sequencing (bulkseq) datasets and mapped these gene expression profiles onto three T2D single-cell RNA sequencing (scRNAseq) datasets.
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