Background: Classic congenital adrenal hyperplasia, primarily due to 21-hydroxylase deficiency, leads to impaired cortisol and aldosterone production and excess adrenal androgens. Lifelong glucocorticoid therapy is required, often necessitating supraphysiological doses in youth to manage androgen excess and growth acceleration. These patients experience higher obesity rates, hypertension, and glucose metabolism issues, complicating long-term health management.
View Article and Find Full Text PDFHypoxic-ischemic (HI) brain injury is a common neurological problem in neonates. The postsynaptic density protein-95 (PSD-95) is an excitatory synaptic scaffolding protein that regulates synaptic function, and represents a potential therapeutic target to attenuate HI brain injury. Syn3 and d-Syn3 are novel high affinity cyclic peptides that bind the PDZ3 domain of PSD-95.
View Article and Find Full Text PDFBackground: Chronic graft-vs-host disease (cGvHD) is a severe immune-mediated complication that affects patients following allogeneic hematopoietic stem cell transplantation (allo-HSCT). Oral manifestations of cGvHD, such as ulcers and mucosal inflammation, significantly impair quality of life and often require long-term treatment. Existing therapies provide limited relief, prompting the exploration of new approaches, including the use of autologous platelet lysate (PL) gel for its regenerative properties.
View Article and Find Full Text PDFBackground: Bariatric surgery is the most effective intervention for severe pediatric obesity, but a subset of youth experience suboptimal weight loss and/or recurrent weight gain. Early re-initiation of obesity pharmacotherapy postoperatively may improve outcomes, though this has not been evaluated in pediatric populations.
Methods: A retrospective cohort study at a tertiary care children's hospital evaluated the safety and efficacy of reintroducing obesity pharmacotherapy within six weeks after laparoscopic sleeve gastrectomy (LSG).
Everolimus, an oral inhibitor of the mammalian target of rapamycin (mTOR), is actually used to prevent organ transplant rejection and treat metastatic breast, renal, and neuroendocrine cancers. Despite significant pharmacokinetic variability among patients, routine therapeutic drug monitoring (TDM) is not commonly used in oncology. The aim of this multicenter, prospective observational cohort study is to assess the prevalence of everolimus minimum concentration at a steady state (Cminss) falling outside the therapeutic range (10-26.
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