keyword
https://read.qxmd.com/read/38680552/dose-response-evaluation-of-intravenous-gene-therapy-in-a-symptomatic-mouse-model-of-metachromatic-leukodystrophy
#41
JOURNAL ARTICLE
Emilie Audouard, Nicolas Khefif, Charlotte Mansat, Océane Nelcha, Elena-Gaia Banchi, Camille Lupiet, Dominique Farabos, Antonin Lamaziere, Caroline Sevin, Françoise Piguet
Metachromatic leukodystrophy (MLD) is a rare, autosomal recessive neurodegenerative disease caused by deficient activity of the lysosomal enzyme arylsulfatase A (ARSA), resulting in sulfatide accumulation and subsequent demyelination and neuronal damage within the central and peripheral nervous systems. Three clinical forms of MLD have been described, based on age at symptom onset. The most frequent and severe forms have an early onset, with the disease progressing rapidly toward severe motor and cognitive regression and ultimately premature death...
June 13, 2024: Molecular Therapy. Methods & Clinical Development
https://read.qxmd.com/read/38680528/paracentral-acute-middle-maculopathy-in-a-patient-with-myelin-oligodendrocyte-glycoprotein-antibody-associated-optic-neuritis
#42
Aniruddh Heroor, Mudit Tyagi, Ramesh Kekunnaya, Goura Chattannavar
PURPOSE: There is insufficient literature reporting the concurrent occurrence of retinal ischemic lesions with optic neuritis. In this case report, we present a distinctive instance of Optic Neuritis with a positive Myelin Oligodendrocyte glycoprotein (MOG) antibody, accompanied by retinal ischemia manifesting as paracentral acute middle maculopathy (PAMM) lesions. OBSERVATIONS: Our patient is a 25-year-old female who tested positive for MOG antibodies and exhibited retinal PAMM lesions without any apparent underlying ischemic cause...
June 2024: American Journal of Ophthalmology Case Reports
https://read.qxmd.com/read/38680525/late-onset-pseudomonas-aeruginosa-orbital-cellulitis-following-glaucoma-drainage-device-implantation
#43
Shayma Jawad, Kevin Halenda
PURPOSE: To present a rare case of late-onset Pseudomonas aeruginosa orbital cellulitis following glaucoma drainage device (GDD) implantation due to suture erosion. OBSERVATIONS: A 65-year-old male with a history of aphakic glaucoma and two remote prior glaucoma drainage device (GDD) surgeries of the right eye presented with right orbital signs. On examination, exposed securing Gore-Tex suture material over the plate of a GDD in the inferotemporal quadrant was present...
June 2024: American Journal of Ophthalmology Case Reports
https://read.qxmd.com/read/38680207/developing-a-remotely-delivered-intensive-outpatient-program-adapted-for-hospitalized-patients-with-opioid-use-disorder-a-qualitative-study
#44
JOURNAL ARTICLE
Veronica Szpak, Sara Prostko, Roger D Weiss, Joji Suzuki
INTRODUCTION: Individuals with opioid use disorder (OUD) who inject drugs have an elevated risk of experiencing serious injection-related infections. While such infections can be treated, treatment for the underlying OUD is often limited. One potential strategy for more intensive addiction treatment is to offer a remotely delivered intensive outpatient program (IOP), adapted from an existing remote IOP ("Smart IOP"). We aimed to conduct a qualitative study to gather feedback on Smart IOP and identify adaptations needed for hospitalized patients...
June 2024: Addictive Behaviors Reports
https://read.qxmd.com/read/38679727/safety-and-efficacy-of-umbilical-cord-tissue-derived-mesenchymal-stem-cells-in-the-treatment-of-patients-with-aging-frailty-a-phase-i-ii-randomized-double-blind-placebo-controlled-study
#45
RANDOMIZED CONTROLLED TRIAL
Yingqian Zhu, Ce Huang, Liang Zheng, Qingqing Li, Jianli Ge, ShaSha Geng, Miaomiao Zhai, Xin Chen, Huixiao Yuan, Yang Li, Wenwen Jia, Keping Sun, Yan Li, Tong Ye, Zhengmei Zhao, Hailiang Liu, Zhongmin Liu, Hua Jiang
BACKGROUND: Mesenchymal stem cells (MSCs) hold a great promise for cell-based therapy in the field of regenerative medicine. In this study, we aimed to evaluate the safety and efficacy of intravenous infusion of human umbilical cord-derived MSCs (HUC-MSCs) in patients with aging frailty. METHODS: In this randomized, double-blind, placebo-controlled trial, participants diagnosed with aging frailty were randomly assigned to receive intravenous administrations of HUC-MSCs or placebo...
April 29, 2024: Stem Cell Research & Therapy
https://read.qxmd.com/read/38679687/aso-author-reflections-surgical-management-of-wilms-tumors-with-intravenous-extension-a-multicenter-analysis-of-clinical-management-with-technical-insights
#46
JOURNAL ARTICLE
Luca Pio, Simone Abib, Florent Guerin, Christophe Chardot, Thomas Blanc, Nadia Sarrai, Helene Martelli, Fernanda K M de Souza, Mayara C A Fanelli, Daniel Tamisier, José Cícero S Guilhen, Emmanuel Le Bret, Emré Belli, Elie Fadel, Monica D S Cypriano, Véronique Minard, Claudia Pasqualini, Gudrun Schleiermacher, Lauriane Lemelle, Julien Rod, Sabine Irtan, Angela Pistorio, Frederic Gauthier, Sophie Branchereau, Sabine Sarnacki
No abstract text is available yet for this article.
April 28, 2024: Annals of Surgical Oncology
https://read.qxmd.com/read/38679534/-translated-article-retrospective-study-of-home-antibiotic-infusion-therapy-using-elastomeric-infusion-pumps
#47
JOURNAL ARTICLE
Sara Ferro Rodríguez, Yelco Chantres Legaspi, Eva María Romay Lema, Blanca Ayuso García, Paloma Castellano Copa, Pedro Peinó Camba, Andrea Barcia Losada, Cristina Rodríguez Díaz
OBJECTIVES: To describe the experience of home antibiotic infusion therapy using elastomeric infusion pumps, administered to patients admitted to the Home Hospitalisation Unit of a tertiary hospital for 3 years and to analyse clinical evolution and mortality. METHOD: Retrospective observational study. The medical history of the patients included in the study was reviewed. Information was obtained on personal history, antimicrobial therapy received, and clinical evolution...
April 27, 2024: Farmacia Hospitalaria
https://read.qxmd.com/read/38679455/does-perioperative-hydrocortisone-or-indomethacin-improve-pancreatoduodenectomy-outcomes-a-triple-arm-randomized-placebo-controlled-trial
#48
JOURNAL ARTICLE
Kislay Kant, Zeeshan Ahmed, Rohit Dama, Monish Karunakaran, Prateek Arora, Pradeep Rebala, Guduru Venkat Rao
BACKGROUNDS/AIMS: This trial evaluated whether anti-inflammatory agents hydrocortisone (H) and indomethacin (I) could reduce major complications after pancreatoduodenectomy (PD). METHODS: Between June 2018 and June 2020, 105 patients undergoing PD with > 40% of acini on the intraoperative frozen section were randomized into three groups (35 patients per group): 1) intravenous H 100 mg 8 hourly, 2) rectal I suppository 100 mg 12 hourly, and 3) placebo (P) from postoperative day (POD) 0-2...
April 29, 2024: Annals of Hepato-Biliary-Pancreatic Surgery
https://read.qxmd.com/read/38679336/-ultra-long-fviii-a-major-step-forward-to-a-hemophilia-free-mind
#49
REVIEW
Cedric Hermans, Glenn F Pierce
A remarkable step forward in the treatment of hemophilia A has recently been achieved with the development of an Ultra-Long modified FVIII. Leveraging expertise gained with fusion to immunoglobulin Fc fragments, disconnecting FVIII from endogenous von Willebrand factor (via a D'-D3 fragment), and benefiting from the pharmacokinetic prolongation provided by the addition of hydrophilic polypeptides, efanesoctocog alfa opens a new era in the treatment of hemophilia A. The term Ultra-Long (UL) FVIII has been proposed to designate it and differentiate it from EHL FVIII...
April 26, 2024: Journal of Thrombosis and Haemostasis: JTH
https://read.qxmd.com/read/38679232/a-review-on-commercial-oligonucleotide-drug-products
#50
REVIEW
Bhavani Prasad Vinjamuri, Jiayi Pan, Paul Peng
Oligonucleotide drug products commercially approved in the US and the EU are reviewed. A total of 20 products that includes 1 aptamer, 12 antisense oligonucleotides (ASOs), 6 small interfering ribonucleic acids (siRNAs), and 1 mixture of single-stranded and double-stranded polydeoxyribonucleotides have been identified. A typical oligonucleotide formulation is composed of an oligonucleotide with buffering agent(s), pH adjusting agents, and a tonicity adjusting agent. All the products are presented as 2.1 - 200 mg/mL solutions at pH between 6 and 8...
April 26, 2024: Journal of Pharmaceutical Sciences
https://read.qxmd.com/read/38679231/blood-to-testis-transport-of-ribavirin-involves-carrier-mediated-processes-at-the-blood-testis-barrier
#51
JOURNAL ARTICLE
Takeru Ito, Yoshiyuki Kubo, Yuma Tega, Shin-Ichi Akanuma, Ken-Ichi Hosoya
Ribavirin, an antiretroviral agent targeting the hepatitis C virus, causes male reproductive toxicity. This study investigated the mechanism of ribavirin transport at the blood-testis barrier (BTB). In vivo mouse integration plot analysis after intravenous administration revealed that the net influx clearance of [3 H]ribavirin in the testis was 3.6-fold greater than that of [14 C]D-mannitol, a paracellular transport marker, implying transcellular transport of ribavirin across the BTB. Moreover, [3 H]ribavirin uptake by TM4 cells, mouse-derived Sertoli cells, was time- and concentration-dependent, with a Km value of 2...
April 26, 2024: Journal of Pharmaceutical Sciences
https://read.qxmd.com/read/38679213/development-of-a-topical-treatment-for-tegumentary-leishmaniasis-using-8-hydroxyquinoline
#52
JOURNAL ARTICLE
Naianda Rezende Ribeiro, Lucas Magno Oliveira Santos, Scarleth Silva Costa, Carolina Kei Myasaki, Daniela Pagliara Lage, Vivian Tamietti Martins, Rory Cristiane Fortes De Brito, Larissa Chaves Freire, Ana Maria Ravena Severino Carvalho, Bryan Victor Serafim Brigido, Ricardo José Alves, Eduardo Antonio Ferraz Coelho, Bruno Mendes Roatt, Daniel Menezes-Souza, Mariana Costa Duarte
In the context of neglected diseases, tegumentary leishmaniasis (TL) presents an emerging and re-emerging character in the national territory and in the world. The treatment of TL has limitations, such as intravenous administration route, high toxicity, and high treatment costs. Thus, several researchers work on new therapeutic strategies to improve the effectiveness of the treatment of leishmaniasis. In this light, the present study used a topical formulation, containing 8-hydroquinoline (8-HQN), for the treatment of Balb/c mice infected with L...
April 26, 2024: European Journal of Pharmaceutics and Biopharmaceutics
https://read.qxmd.com/read/38679076/therapeutic-efficacy-of-adrenergic-agents-on-systemic-and-spinal-hemodynamics-in-an-acute-cervical-spinal-cord-injury-rodent-model
#53
JOURNAL ARTICLE
Kun-Ze Lee, Tzu-Ting Liu, Rui-Yi Chen
BACKGROUND: Cervical spinal cord injury usually results in cardiorespiratory dysfunctions due to interruptions of the bulbospinal pathways innervating the cervical phrenic motoneurons and thoracic sympathetic preganglionic neurons. PURPOSE: The present study aimed to evaluate the therapeutic effects of adrenergic agents on systemic and spinal hemodynamics during acute cervical spinal cord injury. STUDY DESIGN: In vivo animal study. METHODS: The cardiorespiratory function and spinal cord blood flow and oxygenation level were monitored in response to cervical spinal cord contusion and intravenous infusion of three types of adrenergic agents (phenylephrine, dobutamine, and norepinephrine)...
April 26, 2024: Spine Journal: Official Journal of the North American Spine Society
https://read.qxmd.com/read/38679042/azithromycin-therapy-for-prevention-of-chronic-lung-disease-of-prematurity-aztec-a-multicentre-double-blind-randomised-placebo-controlled-trial
#54
JOURNAL ARTICLE
John Lowe, David Gillespie, Ali Aboklaish, Tin Man Mandy Lau, Claudia Consoli, Malavika Babu, Mark Goddard, Kerenza Hood, Nigel Klein, Emma Thomas-Jones, Mark Turner, Marie Hubbard, Julian Marchesi, Janet Berrington, Sailesh Kotecha
BACKGROUND: Systematic reviews have reported conflicting evidence on whether macrolide antibiotics reduce rates of chronic lung disease of prematurity (CLD) in at-risk preterm infants born at less than 30 weeks' gestation, including in those colonised with pulmonary Ureaplasma spp. Since an adequately powered trial has been lacking, we aimed to assess if the macrolide azithromycin improved survival without the development of physiologically defined moderate or severe CLD in preterm infants...
April 25, 2024: Lancet Respiratory Medicine
https://read.qxmd.com/read/38678444/antibiotic-route-and-outcomes-for-children-hospitalized-with-pneumonia
#55
JOURNAL ARTICLE
Jillian M Cotter, Mathew Hall, Mark I Neuman, Anne J Blaschke, Thomas V Brogan, Jonathan D Cogen, Jeffrey S Gerber, Adam L Hersh, Susan C Lipsett, Daniel J Shapiro, Lilliam Ambroggio
BACKGROUND: Emerging evidence suggests that initial oral and intravenous (IV) antibiotics have similar efficacy in pediatric community-acquired pneumonia (CAP), but further data are needed. OBJECTIVE: We determined the association between hospital-level initial oral antibiotic rates and outcomes in pediatric CAP. DESIGNS, SETTINGS AND PARTICIPANTS: This retrospective cohort study included children hospitalized with CAP at 43 hospitals in the Pediatric Health Information System (2016-2022)...
April 28, 2024: Journal of Hospital Medicine: An Official Publication of the Society of Hospital Medicine
https://read.qxmd.com/read/38678266/barriers-to-access-to-hepatitis-c-treatment-with-direct-acting-antivirals-in-people-who-inject-drugs-in-the-community-setting
#56
JOURNAL ARTICLE
Elena Yela, Neus Solé, Lidia Puig, Darío López Gallegos, Rafael Clua-García
Barriers to access to hepatitis C treatment with direct-acting antivirals in people who inject drugs in the community setting. Qualitative study with prison population. Hepatitis C (HCV) treatments with direct-acting antiviral therapy (DAA) are an easy and effective option among people who inject drugs (PWID). However, difficulties in accessing and monitoring treatment in community services and dropouts on release from prison are detected among PWID. For this reason, the aim of the study is to know the access barriers in the diagnosis and treatment of HCV in community health services...
April 27, 2024: Harm Reduction Journal
https://read.qxmd.com/read/38678209/lidocaine-effects-on-neutrophil-extracellular-trapping-and-angiogenesis-biomarkers-in-postoperative-breast-cancer-patients-with-different-anesthesia-methods-a-prospective-randomized-trial
#57
RANDOMIZED CONTROLLED TRIAL
Wenjuan Zhang, Jiao Liu, Xiaohui Li, Zhixia Bai, Yan Sun, Xuexin Chen
BACKGROUND: Anesthesia techniques and drug selection may influence tumor recurrence and metastasis. Neutrophil extracellular trapping (NETosis), an immunological process, has been linked to an increased susceptibility to metastasis in individuals with tumors. Furthermore, recurrence may be associated with vascular endothelial growth factor A (VEGF-A), a mediator of angiogenesis. This study investigates the impact of lidocaine (combined with sevoflurane or propofol anesthesia ) during breast cancer surgery inhibits the expression of biomarkers associated with metastasis and recurrence (specifically H3Cit, NE, MPO, MMP-9 and VEGF-A)...
April 27, 2024: BMC Anesthesiology
https://read.qxmd.com/read/38677720/safety-and-tolerability-of-op-724-in-patients-with-haemophilia-and-liver-cirrhosis-due-to-hiv-hcv-coinfection-an-investigator-initiated-open-label-non-randomised-single-centre-phase-i-study
#58
JOURNAL ARTICLE
Kiminori Kimura, Junko Tanuma, Masamichi Kimura, Jun Imamura, Mikio Yanase, Ichiro Ieiri, Masayuki Kurosaki, Tsunamasa Watanabe, Tomoyuki Endo, Hiroshi Yotsuyanagi, Hiroyuki Gatanaga
OBJECTIVE: Patients with haemophilia and HIV who acquire hepatitis C virus (HCV) after receiving contaminated blood products can experience accelerated progression of liver fibrosis and a poor prognosis, making liver disease a prominent cause of mortality among these patients. In the current study, we aimed to evaluate the safety and tolerability of the potential antifibrotic agent OP-724-a CREB-binding protein/β-catenin inhibitor-in this patient subset. DESIGN: In this single-centre, open-label, non-randomised, phase I trial, we sequentially enrolled patients with cirrhosis following HIV/HCV coinfection classified as Child-Pugh (CP) class A or B...
April 27, 2024: BMJ Open Gastroenterology
https://read.qxmd.com/read/38677443/pharmaceutical-and-biotech-industry-perspectives-on-optimizing-patient-experience-and-treatment-adherence-through-subcutaneous-drug-delivery-design
#59
REVIEW
Jennifer Stevenson, Rachel Poker, Johanna Schoss, Michael Campbell, Claire Everitt, Brian Holly, Nicholas Stones, Ronald J Pettis, Manuel Sanchez-Felix
Subcutaneous (SC) drug delivery can be a safe, effective alternative to the traditional intravenous route of administration, potentially offering notable advantages for both patients and healthcare providers. The SC Drug Development & Delivery Consortium convened in 2018 to raise awareness of industry challenges to advance the development of patient-centric SC drug delivery strategies. The SC Consortium identified better understanding of patient preferences and perspectives as necessary to optimize SC product design attributes and help guide design decisions during SC product development...
April 25, 2024: Advanced Drug Delivery Reviews
https://read.qxmd.com/read/38677347/perioperative-cefazolin-for-total-joint-arthroplasty-patients-who-have-a-penicillin-allergy-is-it-safe
#60
JOURNAL ARTICLE
Brandon R Bukowski, Ricardo J Torres-Ramirez, Daniel Devine, Yu-Fen Chiu, Alberto V Carli, Daniel B Maalouf, Enrique A Goytizolo, Andy O Miller, Jose A Rodriguez
BACKGROUND: Cefazolin is the standard of care for perioperative antibiotic prophylaxis in total joint arthroplasty (TJA) in the United States. The potential allergic cross-reactivity between cefazolin and penicillin causes uncertainty regarding optimal antibiotic choice in patients who have a reported penicillin allergy (rPCNA). The purpose of this study was to determine the safety of perioperative cefazolin in PCNA patients undergoing primary TJA. METHODS: We identified all patients (n = 49,842) undergoing primary total hip arthroplasty (n = 25,659) or total knee arthroplasty (n = 24,183) from 2016 to 2022 who received perioperative intravenous antibiotic prophylaxis...
April 25, 2024: Journal of Arthroplasty
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