keyword
https://read.qxmd.com/read/38537061/cost-effectiveness-of-bevacizumab-therapy-in-the-care-of-patients-with-hereditary-hemorrhagic-telangiectasia
#1
JOURNAL ARTICLE
Daniel Wang, Satoko Ito, Christina Waldron, Ayesha Butt, Ellen Zhang, Harlan Krumholz, Hanny Al-Samkari, George Goshua
No FDA or EMA approved therapies exist for bleeding due to hereditary hemorrhagic telangiectasia (HHT), the second-most-common inherited bleeding disorder worldwide. The current standard-of-care (SOC) includes iron and red cell supplementation, alongside the necessary hemostatic procedures, none of which target underlying disease pathogenesis. Recent evidence has demonstrated that bleeding pathophysiology is amenable to systemic antiangiogenic therapy with the anti-VEGF bevacizumab. Despite its high cost, the addition of longitudinal bevacizumab to the current SOC may reduce overall healthcare resource utilization and improve patient quality-of-life...
March 27, 2024: Blood Advances
https://read.qxmd.com/read/38536458/scabies-in-infants-and-children-a-narrative-review
#2
REVIEW
Anca E Chiriac, Adriana Diaconeasa, Raluca Miulescu, Uwe Wollina
Scabies is a human ectoparasitosis caused by Sarcoptes scabei var. hominis. World-wide around 300 million patients are affected. Infants and children have the highest incidence rates. Poverty and overcrowding are social factors contributing to a higher risk of transmission and treatment failure. The leading symptom of the infestation is itch. Complications are bacterial infections that are responsible for mortality. Diagnosis is clinical. Non-invasive imaging technologies like dermoscopy can be used. Polymerase chain reaction (PCR) is less sensitive and specific than microscopy of skin scrapings...
March 27, 2024: European Journal of Pediatrics
https://read.qxmd.com/read/38535119/adme-gene-related-pharmacogenomic-labeling-of-fda-approved-drugs-comparison-with-clinical-pharmacogenetics-implementation-consortium-cpic-evidence-levels
#3
JOURNAL ARTICLE
Subrata Deb, Robert Hopefl, Anthony Allen Reeves, Dena Cvetkovic
Pharmacogenomics (PGx) can facilitate the transition to patient-specific drug regimens and thus improve their efficacy and reduce toxicity. The aim of this study was to evaluate the overlap of PGx classification for drug absorption, distribution, metabolism, and elimination (ADME)-related genes in the U.S. Food and Drug Administration (FDA) PGx labeling and in the Clinical Pharmacogenetics Implementation Consortium (CPIC) database. FDA-approved drugs and PGx labeling for ADME genes were identified in the CPIC database...
February 20, 2024: Medicines (Basel, Switzerland)
https://read.qxmd.com/read/38534700/the-clinical-efficacy-of-adding-ceftazidime-avibactam-to-standard-therapy-in-treating-infections-caused-by-carbapenem-resistant-klebsiella-pneumonia-with-blaoxa-48-like-genes
#4
JOURNAL ARTICLE
Al Maamon R Abu Jaber, Bilgen Basgut, Ali Abdullah Hawan, Ali Amer Al Shehri, Sultan Ahmad AlKahtani, Nehad J Ahmed, Abdikarim Abdi
Ceftazidime/avibactam (CAZ-AVI) is FDA-approved for managing infections caused by resistant gram-negative bacilli, particularly infections via carbapenem-resistant Enterobacterales pathogens. The clinical data are still limited, particularly those in Saudi Arabia. The present study is a retrospective cohort study that was carried out at the Armed Forces Hospital in the southern region of Saudi Arabia to compare the clinical and microbiological outcomes for CAZ-AVI-treated patients as monotherapy and as an add-on to standard therapy for carbapenem-resistant Klebsiella pneumonia (CRKP) OXA-48 infections to those treated with standard drugs...
March 16, 2024: Antibiotics
https://read.qxmd.com/read/38533505/ozanimod-mediated-remission-in-experimental-autoimmune-encephalomyelitis-is-associated-with-enhanced-activity-of-cns-cd27-low-nk-cell-subset
#5
JOURNAL ARTICLE
Doua Kamyan, Maya Hassane, Alanood Alnaqbi, Abdul-Kader Souid, Zakeya Al Rasbi, Abeer Al Tahrawi, Mariam Al Shamsi
BACKGROUND: Ozanimod (RPC1063) is an immunomodulator that has been recently approved by the FDA (2020) for the treatment of relapsing-remitting multiple sclerosis (RRMS). It is a selective agonist of the sphingosine-1-phophate receptors 1 and 5, expressed on naïve and central memory T and B cells, as well as natural killer (NK) cells, and is involved in lymphocyte trafficking. Oral administration of ozanimod was reported to result in rapid and reversible reduction in circulating lymphocytes in multiple sclerosis (MS) patients, however, only minimal effect on NK cells was observed...
2024: Frontiers in Immunology
https://read.qxmd.com/read/38532266/a-gastroenterologist-s-guide-to-drug-interactions-of-small-molecules-for-inflammatory-bowel-disease
#6
REVIEW
Sivan Harnik, Bella Ungar, Ronen Loebstein, Shomron Ben-Horin
Small molecule drugs are becoming increasingly used in the treatment of inflammatory bowel diseases (IBD). However, unlike monoclonal antibody drugs, which have few interactions with other medications, the pharmacokinetics of small molecule drugs are complex and may be influenced by a myriad of drug-drug interactions (DDI) as well as by patient characteristics and food intake. This review aims to provide a concise practical guide to small molecule drug interactions for the use of IBD physicians. It starts with a brief overview of the main metabolizing enzymes and transporters involved in drug interactions and the Food and Drug Administration's (FDA) approach to determining drug-interaction hazard thresholds...
March 26, 2024: United European Gastroenterology Journal
https://read.qxmd.com/read/38532246/benzopyrone-a-privileged-scaffold-in-drug-discovery-an-overview-of-fda-approved-drugs-and-clinical-candidates
#7
REVIEW
Venu Sharma, Ankita Sharma, Bhagyashri N Wadje, Sandip B Bharate
Natural products have always served as an important source of drugs for treating various diseases. Among various privileged natural product scaffolds, the benzopyrone class of compounds has a substantial presence among biologically active compounds. One of the pioneering anticoagulant drugs, warfarin approved in 1954 bears a benzo-α-pyrone (coumarin) nucleus. The widely investigated psoriasis drugs, methoxsalen, and trioxsalen, also contain a benzo-α-pyrone nucleus. Benzo-γ-pyrone (chromone) containing drugs, cromoglic acid, and pranlukast were approved as treatments for asthma in 1982 and 2007, respectively...
March 26, 2024: Medicinal Research Reviews
https://read.qxmd.com/read/38532176/benchmarking-ani-potentials-as-a-rescoring-function-and-screening-fda-drugs-for-sars-cov-2-m-pro
#8
JOURNAL ARTICLE
Irem N Zengin, M Serdar Koca, Omer Tayfuroglu, Muslum Yildiz, Abdulkadir Kocak
Here, we introduce the use of ANI-ML potentials as a rescoring function in the host-guest interaction in molecular docking. Our results show that the "docking power" of ANI potentials can compete with the current scoring functions at the same level of computational cost. Benchmarking studies on CASF-2016 dataset showed that ANI is ranked in the top 5 scoring functions among the other 34 tested. In particular, the ANI predicted interaction energies when used in conjunction with GOLD-PLP scoring function can boost the top ranked solution to be the closest to the x-ray structure...
March 27, 2024: Journal of Computer-aided Molecular Design
https://read.qxmd.com/read/38532028/pdgfra-k385-mutants-in-myxoid-glioneuronal-tumors-promote-receptor-dimerization-and-oncogenic-signaling
#9
JOURNAL ARTICLE
Laurence de Villenfagne, Ariane Sablon, Jean-Baptiste Demoulin
Myxoid glioneuronal tumors (MGNT) are low-grade glioneuronal neoplasms composed of oligodendrocyte-like cells in a mucin-rich stroma. These tumors feature a unique dinucleotide change at codon 385 in the platelet-derived growth factor receptor α (encoded by the PDGFRA gene), resulting in the substitution of lysine 385 into leucine or isoleucine. The functional consequences of these mutations remain largely unexplored. Here, we demonstrated their oncogenic potential in fibroblast and Ba/F3 transformation assays...
March 26, 2024: Scientific Reports
https://read.qxmd.com/read/38531888/background-and-clinical-significance-of-biomarker-based-patient-enrichment-in-non-small-cell-lung-cancer-drug-development
#10
JOURNAL ARTICLE
Kenji Harada, Shunsuke Ono
Pharmaceutical companies have adopted biomarker-based enrichment (personalized) strategies to improve research and development productivity. We explored the background in which personalized strategies are adopted and examined whether their adoption is linked to improved efficacy of new drugs approved for non-small cell lung cancer (NSCLC) by US Food and Drug Administration (FDA). We extracted data from the first labels of drugs approved for NSCLC between May 2003 and February 2021, and performed a qualitative comparative analysis and meta-analysis...
March 26, 2024: Scientific Reports
https://read.qxmd.com/read/38531819/a-mechanistic-and-preclinical-assessment-of-biorestore-bioactive-glass-as-a-synthetic-bone-graft-extender-and-substitute-for-osteoinduction-and-spine-fusion
#11
JOURNAL ARTICLE
Elianna J Fred, Silvia Minardi, Alyssa M Goodwin, Tejas S Nandurkar, Mark A Plantz, Joseph G Lyons, Jonathan T Paul, James P Foley, Allison J Wintring, Andrew A Furman, Soyeon Jeong, Chawon Yun, Stuart R Stock, Wellington K Hsu, Erin L Hsu
STUDY DESIGN: Preclinical animal study. OBJECTIVE: Evaluate the osteoinductivity and bone regenerative capacity of BioRestore bioactive glass. SUMMARY OF BACKGROUND DATA: BioRestore is a Food and Drug Administration (FDA)-approved bone void filler that has not yet been evaluated as a bone graft extender or substitute for spine fusion. METHODS: In vitro and in vivo methods were used to compare BioRestore with other biomaterials for the capacity to promote osteodifferentiation and spinal fusion...
March 22, 2024: Clinical Spine Surgery
https://read.qxmd.com/read/38531038/glp-1-receptor-agonists-a-review-of-glycemic-benefits-and-beyond
#12
JOURNAL ARTICLE
LaDonna Clark
Type 2 diabetes mellitus (T2DM) is a chronic medical condition affecting millions of individuals worldwide. The burden of disease is significant, as demonstrated by high morbidity and mortality and billions of healthcare dollars spent. The pathophysiology of T2DM is complex, with eight primary deficits. In recent years, an increased focus has been placed on incretin hormones, such as glucagon-like peptide-1 (GLP-1) for its glucose-lowering benefits. Several FDA-approved short-acting and long-acting GLP-1 receptor agonists (GLP-1 RAs) are available in the United States for the treatment of T2DM...
April 1, 2024: JAAPA: Official Journal of the American Academy of Physician Assistants
https://read.qxmd.com/read/38530888/normalizing-granuloma-vasculature-and-matrix-improves-drug-delivery-and-reduces-bacterial-burden-in-tuberculosis-infected-rabbits
#13
JOURNAL ARTICLE
Meenal Datta, Laura E Via, Véronique Dartois, Danielle M Weiner, Matthew Zimmerman, Firat Kaya, April M Walker, Joel D Fleegle, Isaac D Raplee, Colton McNinch, Maksym Zarodniuk, Walid S Kamoun, Changli Yue, Ashwin S Kumar, Sonu Subudhi, Lei Xu, Clifton E Barry, Rakesh K Jain
Host-directed therapies (HDTs) represent an emerging approach for bacterial clearance during tuberculosis (TB) infection. While most HDTs are designed and implemented for immuno-modulation, other host targets-such as nonimmune stromal components found in pulmonary granulomas-may prove equally viable. Building on our previous work characterizing and normalizing the aberrant granuloma-associated vasculature, here we demonstrate that FDA-approved therapies (bevacizumab and losartan, respectively) can be repurposed as HDTs to normalize blood vessels and extracellular matrix (ECM), improve drug delivery, and reduce bacterial loads in TB granulomas...
April 2, 2024: Proceedings of the National Academy of Sciences of the United States of America
https://read.qxmd.com/read/38530703/chemical-versatility-in-catalysis-and-inhibition-of-the-class-iib-histone-deacetylases
#14
JOURNAL ARTICLE
David W Christianson
ConspectusThe zinc-dependent histone deacetylases (HDACs 1-11) belong to the arginase-deacetylase superfamily of proteins, members of which share a common α/β fold and catalytic metal binding site. While several HDACs play a role in epigenetic regulation by catalyzing acetyllysine hydrolysis in histone proteins, the biological activities of HDACs extend far beyond histones. HDACs also deacetylate nonhistone proteins in the nucleus as well as the cytosol to regulate myriad cellular processes. The substrate pool is even more diverse in that certain HDACs can hydrolyze other covalent modifications...
March 26, 2024: Accounts of Chemical Research
https://read.qxmd.com/read/38530400/a-year-in-pharmacology-new-drugs-approved-by-the-us-food-and-drug-administration-in-2023
#15
REVIEW
Gizem Kayki-Mutlu, Zinnet Sevval Aksoyalp, Leszek Wojnowski, Martin C Michel
With 54 new drugs and seven cellular and gene therapy products, the approvals by the US Food and Drug Administration (FDA) recovered 2023 from the 2022 dent back to the levels of 2020-2021. As in previous years of this annual review, we assign these new drugs to one of three levels of innovation: first drug against a condition ("first-in-indication"), first drug using a novel molecular mechanism ("first-in-class"), and "next-in-class," i.e., a drug using an already exploited molecular mechanism. We identify four (7%) "first-in-indication," 22 (36%) "first-in-class," and 35 (57%) "next-in-class" drugs...
March 26, 2024: Naunyn-Schmiedeberg's Archives of Pharmacology
https://read.qxmd.com/read/38530160/a-phase-0-1-pharmacokinetic-and-pharmacodynamics-and-safety-and-tolerability-study-of-letrozole-in-combination-with-standard-therapy-in-recurrent-high-grade-gliomas
#16
JOURNAL ARTICLE
Pankaj B Desai, Aniruddha Karve, Misam Zawit, Priyanka Arora, Nimita Dave, Joy Awosika, Ningjing Li, Bethany Fuhrman, Mario Medvedovic, Larry Sallans, Ady Kendler, Biplab DasGupta, David Plas, Richard Curry, Mario Zuccarello, Rekha Chaudhary, Soma Sengupta, Trisha M Wise-Draper
PURPOSE: High grade gliomas (HGGs) carry a poor prognosis, with glioblastoma accounting for almost 50% of primary brain malignancies in the elderly. Unfortunately, despite the use of multiple treatment modalities, the prognosis remains poor in this population. Our pre-clinical studies suggest that the presence of aromatase expression, encoded by CYP19A1, is significantly upregulated in HGGs. Remarkably, we find that letrozole (LTZ), an FDA approved aromatase inhibitor, has marked activity against HGGs...
March 26, 2024: Clinical Cancer Research
https://read.qxmd.com/read/38529911/hematopoietic-cell-kinase-as-a-nexus-for-drug-repurposing-implications-for-cancer-and-hiv-therapy
#17
JOURNAL ARTICLE
Mohammad Ali Abdullah Almoyad, Abdulrhman Alsayari, Shadma Wahab, Subhash Chandra
Hematopoietic cell kinase (HCK) has emerged as a potential target for therapeutic intervention in cancer and HIV infection because of its critical role in critical signaling pathways. Repurposing FDA-approved drugs offers an efficient strategy to identify new treatment options. Here, we address the need for novel therapies in cancer and HIV by investigating the potential of repurposed drugs against HCK. Our goal was to identify promising drug candidates with high binding affinities and specific interactions within the HCK binding pocket...
March 26, 2024: Journal of Biomolecular Structure & Dynamics
https://read.qxmd.com/read/38529713/mechanistic-insights-into-sodium-ion-mediated-ligand-binding-affinity-and-modulation-of-5-ht2b-gpcr-activity-implications-for-drug-discovery-and-development
#18
JOURNAL ARTICLE
Arushi Chauhan, Jitender Singh, Namrata Sangwan, Pramod K Avti
PURPOSE: The G-protein coupled receptor (GPCR) family, implicated in neurological disorders and drug targets, includes the sensitive serotonin receptor subtype, 5-HT2B. The influence of sodium ions on ligand binding at the receptor's allosteric region is being increasingly studied for its impact on receptor structure. METHODS: High-throughput virtual screening of three libraries, specifically the Asinex-GPCR library, which contains 8,532 compounds and FDA-approved (2466 compounds) and investigational compounds (2731)) against the modeled receptor [4IB4-5HT2B RM ] using the standard agonist/antagonist (Ergotamine/Methysergide), as previously selected from our studies based on ADMET profiling, and further on basis of binding free energy a single compound - dihydroergotamine is chosen...
March 26, 2024: Journal of Receptor and Signal Transduction Research
https://read.qxmd.com/read/38529422/advancements-in-mitraclip-intervention-for-mitral-regurgitation-a-comprehensive-review-and-comparative-analysis-of-clinical-trials
#19
REVIEW
Shitij Shrivastava, Shashwat Shrivastava, Sai Vishnu Vardhan Allu, Patrik Schmidt, Moiud Mohyeldin, Abeer Qasim
This comprehensive review explores the evolution and clinical impact of MitraClip intervention in the management of mitral regurgitation. Mitral regurgitation results from dysfunction in the mitral valve (MV) apparatus. The MitraClip Clip Delivery System was approved by the Food and Drug Administration (FDA) in 2013. The discussion delves into the procedural foundation of MitraClip intervention, primarily based on Alfieri's technique of edge-to-edge leaflet approximation. As highlighted by key clinical trials, including Endovascular Valve Edge-to-Edge Repair (EVEREST) II Trial, Cardiovascular Outcomes Assessment of the MitraClip Percutaneous Therapy for Heart Failure Patients with Functional Mitral Regurgitation (COAPT) Trial, and Percutaneous Repair with the MitraClip Device for Severe Functional/Secondary Mitral Regurgitation (MITRA-FR) trial, the efficacy and safety of MitraClip were evaluated in comparison to surgical interventions and guideline-directed medical therapy...
February 2024: Curēus
https://read.qxmd.com/read/38529070/retrospective-subgroup-analysis-of-fecal-microbiota-live-jslm-rebyota-%C3%A2-administered-by-colonoscopy-under-enforcement-discretion-for-the-prevention-of-recurrent-clostridioides-difficile-infection
#20
JOURNAL ARTICLE
Whitfield L Knapple, David S Yoho, Alexander Sheh, Joan Thul, Paul Feuerstadt
BACKGROUND: Fecal microbiota, live-jslm (RBL; REBYOTA® ), is the first Food and Drug Administration (FDA)-approved, single-dose, rectally administered, microbiota-based live biotherapeutic product for preventing Clostridioides difficile infection (CDI) recurrence. Alternative routes of administration are of clinical interest. OBJECTIVES: Evaluate the safety and efficacy of RBL administration via colonoscopy. DESIGN: Retrospective analysis of electronic medical records of participants administered RBL via colonoscopy under FDA enforcement discretion...
2024: Therapeutic Advances in Gastroenterology
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