10,567,583 results match your criteria therapy

Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.

Neurology 2022 May 26. Epub 2022 May 26.

Friedrich-Baur-Institut, Department of Neurology Klinikum München, München, Germany.

Background And Objectives: Pompe disease is a rare, progressive neuromuscular disorder caused by deficiency of lysosomal acid α-glucosidase (GAA) and subsequent glycogen accumulation. Avalglucosidase alfa, a recombinant human GAA enzyme replacement therapy designed for increased cellular uptake and glycogen clearance, has been studied for long-term efficacy and safety in patients with late-onset Pompe disease (LOPD). Here we report up to 6. Read More

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Genome-wide Association and Meta-analysis of Age-at-Onset in Parkinson Disease: Evidence From COURAGE-PD Consortium.

Neurology 2022 May 26. Epub 2022 May 26.

Sorbonne Université (SU) Unité Mixte de Recherche (UMR) 1127, INSERM U1127, CNRS UMR 7225, Institut du Cerveau et de la Moelle épinière, ICM, Paris, France.

Background And Objectives: Considerable heterogeneity exists in the literature concerning genetic determinants of the age of onset (AAO) of Parkinson's disease (PD), which could be attributed to lack of well-powered replication cohorts. The previous largest GWAS identified nd loci on chromosome (Chr) 4 with a significant influence on AAO of PD, these have not been independently replicated. The present study aims to conduct a meta-analysis of GWAS of PD AAO and validate previously observed findings in worldwide populations. Read More

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Radiologic Evaluation Criteria for Chronic Subdural Hematomas: Recommendations for Clinical Trials.

AJNR Am J Neuroradiol 2022 May 26. Epub 2022 May 26.

From the Department of Diagnostic and Interventional Neuroradiolog (R.M., M.B., J.F., U.Z., S.G.), University Medical Center Hamburg-Eppendorf, Hamburg, Germany

Embolization of the middle meningeal artery has gained substantial interest as a therapy for chronic subdural hematomas. For the results of the currently running chronic subdural hematoma trials to inform clinical practice, sufficient accuracy and matching definitions are necessary. We summarized the current practice in chronic subdural hematoma evaluation and derived suggestions on reporting standards using the {Nested} Knowledge AutoLit living review platform. Read More

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Application of 7T MRS to High-Grade Gliomas.

AJNR Am J Neuroradiol 2022 May 26. Epub 2022 May 26.

From the Department of Neurosurgery (L.M., C.G.H.), Icahn School of Medicine at Mount Sinai, Mount Sinai Health System, New York, New York.

MRS, including single-voxel spectroscopy and MR spectroscopic imaging, captures metabolites in high-grade gliomas. Emerging evidence indicates that 7T MRS may be more sensitive to aberrant metabolic activity than lower-field strength MRS. However, the literature on the use of 7T MRS to visualize high-grade gliomas has not been summarized. Read More

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Pro- and anti-fibrotic effects of vascular endothelial growth factor in chronic kidney diseases.

Ren Fail 2022 Dec;44(1):881-892

Department of Nephrology, The First Hospital of Jilin University, Changchun, People's Republic of China.

Renal fibrosis is the inevitable common end-point of all progressive chronic kidney diseases. The underlying mechanisms of renal fibrosis are complex, and currently there is no effective therapy against renal fibrosis. Renal microvascular rarefaction contributes to the progression of renal fibrosis; however, an imbalance between proangiogenic and antiangiogenic factors leads to the loss of renal microvasculature. Read More

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December 2022

Transplantation in the Age of Precision Medicine: The Emerging Field of Treg Therapy.

Semin Nephrol 2022 Jan;42(1):76-85

Center for Transplantation Sciences, Department of Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA; Division of Transplantation, Department of Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA. Electronic address:

Solid organ transplantation has experienced incredible success over the past half century in rescuing patients with end-stage organ failure and bestowing them with a high quality of life. This success in large part is the result of advances in immunosuppression, however, these regimens come with significant costs including opportunistic infections, and in some cases, direct toxicity to the newly transplanted organs. Further advances are needed. Read More

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January 2022

Emerging Concepts in Managing Malignancy in Kidney Transplant Patients.

Semin Nephrol 2022 Jan;42(1):63-75

Transplant Research Center, Renal Division, Brigham and Women's Hospital, Boston, Massachusetts. Electronic address:

Cancer remains a significant cause of morbidity and mortality in kidney transplant recipients, due to long-term immunosuppression. Salient issues to consider in decreasing the burden of malignancy among kidney transplant recipients include pretransplant recipient evaluation, post-transplant screening and monitoring, and optimal treatment strategies for the kidney transplant recipients with cancer. In this review, we address cancer incidence and outcomes, approaches to cancer screening and monitoring pretransplant and post-transplant, as well as treatment strategies, immunosuppressive management, and multidisciplinary approaches in the kidney transplant recipients with cancer. Read More

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January 2022

The Complement System in the Modern Era of Kidney Transplantation: Mechanisms of Injury and Targeted Therapies.

Semin Nephrol 2022 Jan;42(1):14-28

Division of Nephrology, Department of Medicine, Boston University School of Medicine, Boston, MA. Electronic address:

Kidney transplantation remains the treatment of choice for patients with end-stage kidney disease. Significant progress has been made over the course of many years to improve both patient and graft outcomes after transplant. Modern immunosuppressive therapy has reduced the rate of acute rejection and resulted in excellent short- and long-term graft survival. Read More

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January 2022

Circadian Cadence and NR1D1 Tune Cardiovascular Disease.

J Am Coll Cardiol 2022 May;79(21):2116-2118

Cardiovascular Research Institute and the Department of Medicine, Cardiology, Icahn School of Medicine at Mount Sinai, New York, New York, USA; Nash Family Department of Neuroscience, Icahn School of Medicine at Mount Sinai, New York, New York, USA. Electronic address: https://twitter.com/Cam_phd.

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Disruption of Circadian Rhythms by Shift Work Exacerbates Reperfusion Injury in Myocardial Infarction.

J Am Coll Cardiol 2022 May;79(21):2097-2115

State Key Laboratory for Oncogenes and Related Genes, Division of Cardiology, Renji Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai Cancer Institute, Shanghai, China. Electronic address:

Background: Shift work is associated with increased risk of acute myocardial infarction (AMI) and worsened prognosis. However, the mechanisms linking shift work and worsened prognosis in AMI remain unclear.

Objectives: This study sought to investigate the impact of shift work on reperfusion injury, a major determinant of clinical outcomes in AMI. Read More

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Non-tuberculous mycobacterial thoracic osteomyelitis in an immunocompetent host: a rare presentation of .

BMJ Case Rep 2022 May 26;15(5). Epub 2022 May 26.

Department of Infectious Diseases, The University of Iowa Hospitals and Clinics, Iowa City, Iowa, USA.

An immunocompetent man in his 40s presented with 3 months of mid-thoracic back pain which progressed to include progressive paraesthesias and lower extremity weakness. Investigations revealed thoracic spine osteomyelitis with signs of cord compression. He underwent neurosurgical intervention, including laminectomy, spinal cord decompression and partial resection of an epidural mass. Read More

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Affective disorders: eliminate WArning signs and REstore functioning-AWARE-a randomised controlled multimodule intervention study, presentation of design and intervention.

BMJ Open 2022 May 26;12(5):e058839. Epub 2022 May 26.

Research Unit, North Zealand Psychiatric Center, Hillerod, Copenhagen, Denmark.

Introduction: Affective disorders are associated with impaired overall functioning and quality of life (QoL). Despite different medical and psychological treatment options, the prognosis remains largely unchanged. Consequently, the field needs new intervention strategies especially targeting patient groups with impaired functioning. Read More

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Remote versus face-to-face home-based exercise programme in people with amyotrophic lateral sclerosis: protocol for a randomised clinical trial.

BMJ Open 2022 May 26;12(5):e056323. Epub 2022 May 26.

Department of Physical Therapy, Federal University of Rio Grande do Norte, Natal, Brazil.

Introduction: Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease with variable and complex clinical manifestations that requires a multidisciplinary approach. However, face-to-face treatment in this population may experience barriers, such as difficulty accessing physical therapists or other professionals. As a result, strategies (eg, telerehabilitation) emerged to facilitate treatment and physical therapy monitoring. Read More

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Optimizing Post-Acute Care Patient Safety: A Scoping Review of Multifactorial Fall Prevention Interventions for Older Adults.

J Appl Gerontol 2022 May 26:7334648221104375. Epub 2022 May 26.

Department of Sociology, 14669California State University Los Angeles, Los Angeles, CA, USA.

Accidental falls are preventable adverse events for older post-acute care (PAC) patients. Yet, due to the functional and medical care needs of this population, there is little guidance to inform multidisciplinary prevention efforts. This scoping review aims to characterize the evidence for multifactorial PAC fall prevention interventions. Read More

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Real-life experience of ledipasvir and sofosbuvir for HCV infected Korean patients: a multicenter cohort study.

Korean J Intern Med 2022 May 27. Epub 2022 May 27.

The Catholic University Liver Research Center, College of Medicine, The Catholic University of Korea, Seoul, Korea.

Background/aims: To evaluate the efficacy and safety of ledipasvir/sofosbuvir (LDV/SOF) therapy in hepatitis C virus (HCV)-infected Korean patients in a real clinical setting.

Methods: A total of 273 patients who received LDV/SOF therapy between May 2016 and February 2021 were consecutively enrolled and analyzed. A per-protocol analysis was performed to evaluate the virologic response. Read More

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Tumor-associated Tregs obstruct antitumor immunity by promoting T cell dysfunction and restricting clonal diversity in tumor-infiltrating CD8+ T cells.

J Immunother Cancer 2022 May;10(5)

Department of Immunology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida, USA

Background: Accumulation of regulatory T cells (Treg) has been described to often correlate with poor prognosis in many solid tumors. How Treg presence impinges on limited functionality and clonal composition of tumor-associated CD8 +T cells has important implications for their therapeutic targeting in the tumor microenvironment. In the present study, we investigated how accumulation of Tregs contributes to T cell dysfunction and clonal constriction of tumor-infiltrating CD8 +T cells. Read More

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Spatial transcriptomics of macrophage infiltration in non-small cell lung cancer reveals determinants of sensitivity and resistance to anti-PD1/PD-L1 antibodies.

J Immunother Cancer 2022 May;10(5)

Department of Medecine, Institut Bergonié, Bordeaux, France

Background: Tumor-associated macrophages (TAMs) having immunosuppressive properties are one of the most abundant immune cells in the tumor microenvironment (TME). Preclinical studies have highlighted the potential role of TAMs in resistance to immune checkpoint blockers (ICBs). Here, we investigated the predictive value of TAM infiltration in patients with non-small cell lung cancer (NSCLC) treated with ICBs and characterized their transcriptomic profiles. Read More

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Bufalin stimulates antitumor immune response by driving tumor-infiltrating macrophage toward M1 phenotype in hepatocellular carcinoma.

J Immunother Cancer 2022 May;10(5)

Institute of Infectious Disease, Shuguang Hospital Affiliated to Shanghai University of Traditional Chinese Medicine, Shanghai, 201203, People's Republic of China

Background: Immunotherapy for hepatocellular carcinoma (HCC) exhibits limited clinical efficacy due to immunosuppressive tumor microenvironment (TME). Tumor-infiltrating macrophages (TIMs) account for the major component in the TME, and the dominance of M2 phenotype over M1 phenotype in the TIMs plays the pivotal role in sustaining the immunosuppressive character. We thus investigate the effect of bufalin on promoting TIMs polarization toward M1 phenotype to improve HCC immunotherapy. Read More

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Efficacy of pembrolizumab in patients with advanced cancer of unknown primary (CUP): a phase 2 non-randomized clinical trial.

J Immunother Cancer 2022 May;10(5)

Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

Background: Cancer of unknown primary (CUP) is an aggressive rare malignancy with limited treatment options. Data regarding clinical activity of immune checkpoint inhibitors in CUP is lacking. Therefore, we evaluated the efficacy of pembrolizumab, a programmed cell death-1 inhibitor, in patients with CUP. Read More

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Design and application of DNA nanostructures for organelle-targeted delivery of anticancer drugs.

Expert Opin Drug Deliv 2022 May 26. Epub 2022 May 26.

Cancer Metastasis Alert and Prevention Center, Fujian Provincial Key Laboratory of Cancer Metastasis Chemoprevention and Chemotherapy, National & Local Joint Biomedical Engineering Research Center on Photodynamic Technologies, State Key Laboratory of Photocatalysis on Energy and Environment, College of Chemistry, Fuzhou University, Fuzhou 305108, China.

Introduction: DNA nanostructures targeting organelles are of great significance for the early diagnosis and precise therapy of human cancers. This review is expected to promote the development of DNA nanostructure-based cancer treatment with organelle-level precision in the future.

Areas Covered: In this review, we introduce the different principles for targeting organelles, summarize the progresses in the development of organelle-targeting DNA nanostructures, highlight their advantages and applications in disease treatment, and discuss current challenges and future prospects. Read More

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Effectiveness of photodynamic therapy with 5-aminolevulinic acid for Bowenoid papulosis: a retrospective study with long-term follow-up.

Photodiagnosis Photodyn Ther 2022 May 23:102918. Epub 2022 May 23.

Department of Dermatology and Venereology, Nanfang Hospital, Southern Medical University, Guangzhou, 510515, China. Electronic address:

Background: Bowenoid papulosis (BP) is a rare infectious skin disease that occurs in sexually active young people. BP is associated with human papillomavirus (HPV)16 and 18 infections, spreads through sexual contact, and tends to become cancerous. Currently, there are different BP treatment methods, and few reports on the efficacy of photodynamic therapy (PDT) with 5-aminolevulinic acid (ALA) (ALA-PDT). Read More

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Early evidence of the artificial transfer/transplant of mitochondria to oocytes and zygotes by MitoCeption.

Mitochondrion 2022 May 23. Epub 2022 May 23.

Instituto de Investigaciones en Biomedicina iBioMed, Universidad San Francisco de Quito USFQ, Quito, Ecuador; Mito-Act Research Consortium, Quito, Ecuador; Escuela de Medicina, Colegio de Ciencias de la Salud COCSA, Universidad San Francisco de Quito USFQ, Quito, Ecuador; Sistemas Médicos SIME, Universidad San Francisco de Quito USFQ, Quito, Ecuador. Electronic address:

Oocytes may carry mutations in their mitochondrial DNA (mtDNA) which affect fertility and embryo development leading to hereditary diseases or rejection. Mitochondrial replacement therapies (MRTs) such as polar body transfer, spindle transfer and pronuclear transfer, aim to change dysfunctional to normal mitochondria inside oocytes and zygotes resulting in healthier offspring. Even with promising results, MRTs techniques are invasive to oocytes and may negatively affect their viability and the success of the procedure. Read More

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The Applications of Plasma Cell-free DNA in Cancer Detection: Implications in the Management of Breast Cancer Patients.

Crit Rev Oncol Hematol 2022 May 23:103725. Epub 2022 May 23.

Department of Surgery, Cedars-Sinai Medical Center, West Hollywood, CA, 90048, USA; Samuel Oschin Comprehensive Cancer Institute and Cedars-Sinai Medical Center, West Hollywood, CA, 90048, USA; Biomedical Sciences, Cedars-Sinai Medical Center, West Hollywood, CA, 90048, USA.

Liquid biopsy probes DNA, RNA, and proteins in body fluids for cancer detection and is one of the most rapidly developing areas in oncology. Tumor-derived DNA (circulating tumor DNA, ctDNA) in the context of cell-free DNA (cfDNA) in blood has been the main target for its potential utilities in cancer detection. Liquid biopsy can report tumor burden in real-time without invasive interventions, and would be feasible for screening tumor types that lack standard-of-care screening approaches. Read More

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Vorinostat combined with Busulfan, Fludarabine, and Clofarabine Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Acute Leukemia: Long-term Study Outcomes.

Transplant Cell Ther 2022 May 23. Epub 2022 May 23.

Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA. Electronic address:

Background: Conditioning regimens play a major role in determining disease outcomes following allogeneic hematopoietic stem cell transplantation (allo-HSCT). The use of intravenous (IV) busulfan (Bu) as part of the conditioning chemotherapy has been shown to be effective in controlling disease relapse. However, disease relapse remains a major cause of death following allo-HSCT. Read More

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Characterization of phospholipid-modified lung surfactant in vitro and in a neonatal ARDS model reveals anti-inflammatory potential and surfactant lipidome signatures.

Eur J Pharm Sci 2022 May 23:106216. Epub 2022 May 23.

Division of Immunobiophysics, Priority Area Infections, Research Center Borstel, Leibniz Lung Center, Borstel, Germany; Kiel Nano, Surface and Interface Science KiNSIS, Kiel University, Germany. Electronic address:

A strong inflammatory immune response drives the lung pathology in neonatal acute respiratory distress syndrome (nARDS). Anti-inflammatory therapy is therefore a promising strategy for improved treatment of nARDS. We demonstrate a new function of the anionic phospholipids POPG, DOPG, and PIP2 as inhibitors of IL-1β release by LPS and ATP-induced inflammasome activation in human monocyte-derived and lung macrophages. Read More

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Amikacin pharmacokinetics in elderly patients with severe infections.

Eur J Pharm Sci 2022 May 23:106219. Epub 2022 May 23.

Hospital Universitario Severo Ochoa, Avenida de Orellana, 28911 Leganés, Spain; Instituto de Investigación Sanitaria Puerta de Hierro - Segovia de Arana, Majadahona, Madrid, Spain. Electronic address:

Objective: The aim of this study was to characterize the population pharmacokinetics of amikacin in elderly patients by means of nonlinear mixed effects modelling and to propose initial dosing schemes to optimize therapy based on PK/PD targets.

Method: A total of 137 elderly patients from 65 to 94 years receiving intravenous amikacin and routine therapeutic drug monitoring at Hospital Universitario Severo Ochoa were included. Concentration-time data and clinical information were retrospectively collected; initial doses of amikacin ranged from 5. Read More

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Fluorinated diselenide nanoparticles for radiosensitizing therapy of cancer.

Free Radic Biol Med 2022 May 23. Epub 2022 May 23.

Institute of Macromolecular Chemistry of the Czech Academy of Sciences, Heyrovsky Sq.2, 162 06, Prague 6, Czech Republic.

Radiation resistance of cancer cells represents one of the major challenges in cancer treatment. The novel self-assembled fluoralkylated diselenide nanoparticles (fluorosomes) based on seleno-l-cystine (17FSe) possess redox-active properties that autocatalytically decompose hydrogen peroxide (HO) and oxidize the intracellular glutathione (GSH) that results in regulation of cellular oxidative stress. Alkylfluorinated diselenide nanoparticles showed a significant cytotoxic and radiosensitizing effect on cancer cells. Read More

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L-Se-methylselenocysteine loaded mucoadhesive thermogel for effective treatment of Vulvar candidiasis.

Int J Pharm 2022 May 23:121851. Epub 2022 May 23.

School of Pharmacy, Health Science Center, Xi'an Jiaotong University, Xi'an, China. Electronic address:

Vulvar candidiasis (VVC) is a vaginitis caused by vaginal mucosa infection of Candida, which greatly impairs women's health. Although there are more and more thiazoles on the market, new classes of antifungal drugs are still missing, it is still challenging to treat azole-resistant candidal vaginitis. We found that L-Se-methylselenocysteine (L-SeMC) could effectively inhibit the growth of Candida albicans, reduce the density and length of the mycelia. Read More

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Development and optimization of sustained-release drug-loaded intravesical inserts via semi-solid micro-extrusion 3D-printing for bladder targeting.

Int J Pharm 2022 May 23:121849. Epub 2022 May 23.

Institute of Pharmaceutics and Biopharmaceutics, Heinrich Heine University Düsseldorf, Universitätsstr. 1, 40225 Düsseldorf, Germany.

Discontinued treatment and non-adherence are oftentimes weaknesses of common first-line drug therapy against bladder conditions due to their negative side-effects. To overcome these limitations and increase patients' quality of life, intravesical therapies are continuously being explored. 3D-printing offers the possibility of freely tailoring drug delivery systems to manufacture indwelling devices that may administer drugs locally over an extended time and avoiding frequently repeated administrations while minimizing systemic side-effects. Read More

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