A5000410098- SARS-CoV-2 and COVID-19 Research
- Hemoglobinopathies and Related Disorders
- CAR-T cell therapy research
- COVID-19 Clinical Research Studies
- Iron Metabolism and Disorders
- Biomedical Ethics and Regulation
- Venous Thromboembolism Diagnosis and Management
- SARS-CoV-2 detection and testing
- Blood groups and transfusion
- Blood donation and transfusion practices
- Acute Myeloid Leukemia Research
- Pluripotent Stem Cells Research
- Vaccine Coverage and Hesitancy
- Long-Term Effects of COVID-19
- COVID-19 epidemiological studies
- Platelet Disorders and Treatments
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Cell Adhesion Molecules Research
- Blood Coagulation and Thrombosis Mechanisms
- Erythrocyte Function and Pathophysiology
- Health Systems, Economic Evaluations, Quality of Life
- Viral Infections and Immunology Research
- Virus-based gene therapy research
- COVID-19 and Mental Health
- Viral Infections and Vectors
UK Health Security Agency
2022-2025
Center for Biologics Evaluation and Research
2013-2024
United States Food and Drug Administration
2014-2024
Cedars-Sinai Medical Center
2024
Lung Institute
2024
St Thomas' Hospital
2024
Wake Forest University
2023
United States Department of Health and Human Services
2023
Department of Health and Social Care
2021
Hellenic Agency for Local Development and Local Government
2021
The FDA is developing guidance on the use of “real-world evidence” — health care information from atypical sources, including electronic records, billing databases, and product disease registries to assess safety effectiveness drugs devices.
BackgroundConvalescent plasma has been widely used to treat coronavirus disease 2019 (Covid-19) under the presumption that such contains potentially therapeutic antibodies severe acute respiratory syndrome 2 (SARS-CoV-2) can be passively transferred recipient. Whether convalescent with high antibody levels rather than low is associated a lower risk of death unknown.MethodsIn retrospective study based on U.S. national registry, we determined anti–SARS-CoV-2 IgG in hospitalized adults...
BACKGROUND. Convalescent plasma is the only antibody-based therapy currently available for patients with coronavirus disease 2019 (COVID-19). It has robust historical precedence and sound biological plausibility. Although promising, convalescent not yet been shown to be safe as a treatment COVID-19.
To ensure that the emerging field of stem-cell therapy fulfills its promise to patients, we must first understand risks and benefits develop therapeutic approaches based on sound science. That requires a commitment principles evidence generation.
As of December 31, 2023, the FDA had become aware 22 cases T-cell cancers that occurred after treatment with CAR-T products. For now, such appear to be relatively rare adverse events.
BackgroundAntigen lateral flow devices (LFDs) have been widely used to control SARS-CoV-2. We aimed improve understanding of LFD performance with changes in variant infections, vaccination, viral load, and use, the detection infectious individuals.MethodsIn this diagnostic study, paired RT-PCR test results were prospectively collected from asymptomatic symptomatic participants UK between Nov 4, 2020, March 21, 2022, support National Health Service (NHS) England's Test Trace programme. The...
This Viewpoint discusses declining vaccination rates in the US, specifically against COVID-19, and ways which clinicians Food Drug Administration can counter current large volume of vaccine misinformation.
Tuberous sclerosis (TSC) is an autosomal dominant genetic disorder in which benign hamartomas develop multiple organs, caused by mutations either TSC1 or TSC2. We developed a murine model of Tsc2 disease using gene targeting approach. Tsc2-null embryos die at embryonic days 9.5-12.5 from hepatic hypoplasia. heterozygotes display 100% incidence bilateral renal cystadenomas, 50% liver hemangiomas, and 32% lung adenomas 15 months age. Progression to carcinoma, fatal bleeding the extremity...
Abstract Defects in myeloid cell function Rac2 knockout mice underline the importance of this isoform activation NADPH oxidase and motility. However, specific role Rac1 neutrophil has been difficult to assess since deletion results embryonic lethality mice. To elucidate neutrophils, we generated with a conditional deficiency restricted cells granulocyte/monocyte lineage. As observed Rac2-deficient Rac1-deficient neutrophils demonstrated profound defects inflammatory recruitment vivo,...
Summary Deferasirox is a once‐daily, oral iron chelator developed for treating transfusional overload. Preclinical studies indicated that the kidney was potential target organ of toxicity. As patients with sickle cell disease often have abnormal baseline renal function, primary objective this randomised, open‐label, phase II trial to evaluate safety and tolerability deferasirox in comparison deferoxamine population. Assessment efficacy, as measured by change liver concentration (LIC) using...
Human neutrophils exhibit multiple increases in cytosolic free calcium concentration [( Ca2+]i) spontaneously and response to the chemoattractant N-formyl-L-methionyl-L-leucyl-L-phenylalanine (Jaconi, M. E. E., R. W. Rivest, Schlegel, C. B. Wollheim, D. Pittet, P. Lew. 1988. J. Biol. Chem. 263:10557-10560). The function of these repetitive [Ca2+]i, as well role Ca2+ human neutrophil migration, remain unresolved. We have used microspectrofluorometry measure [Ca2+]i single fura-2-loaded they...
FDA’s Track for Cell-Based Therapies Added to several other tracks speed development of therapeutic agents that are safe and effective, the FDA has created a new regulatory pathway cell-based therapies in regenerative medicine.
We will address the strategic, organizational, and technical aspects that must be considered through an assessment of current landscape data available to clinicians patients for use in clinical decision making opportunities enhancing body evidence.2. work better identify describe ongoing activities contributing narrowing evidence gap approaches leverage extend volumes relevant digital health care facilitate efficient, streamlined randomized trials high-quality observational studies.3....
Summary Patients with acute myeloid leukaemia ( AML ) or myelodysplastic syndrome MDS may respond to treatment epigenetic‐modifying agents. Histone deacetylase inhibitors synergize hypomethylating This phase 1 dose‐escalation study was designed determine the maximum tolerated dose, recommended 2 safety and tolerability of vorinostat plus decitabine in patients relapsed/refractory , newly‐diagnosed intermediate‐ high‐grade . Thirty‐four received concurrent therapy 37 sequential followed by...
Germline pathogenic variants in the E-cadherin gene (CDH1) are strongly associated with development of hereditary diffuse gastric cancer. There is a paucity data to guide risk assessment and management families cancer that do not carry CDH1 variant, making it difficult make informed decisions about surveillance risk-reducing surgery. We aimed identify new candidate genes predisposition affected without variants.
The United States (US) Expanded Access Program (EAP) to coronavirus disease 2019 (COVID-19) convalescent plasma was initiated in response the rapid spread of severe acute respiratory syndrome 2 (SARS-CoV-2), causative agent COVID-19. While randomized clinical trials were various stages development and enrollment, there an urgent need for widespread access potential therapeutic agents. objective this study is report on demographic, geographical, chronological characteristics patients EAP, key...
Abstract Successful therapeutics and vaccines for coronavirus disease 2019 (COVID-19) have harnessed the immune response to severe acute respiratory syndrome 2 (SARS-CoV-2). Evidence that SARS-CoV-2 exists as locally evolving variants suggests immunological differences may impact effectiveness of antibody-based treatments such convalescent plasma vaccines. Considering near-sourced likely reflects antigenic composition local viral strains, we hypothesize has a higher efficacy, defined by...
This Viewpoint argues that the development of a distinctly improved generation SARS-CoV-2 vaccines is paramount to offering greater breadth and depth protection for longer duration against COVID-19 disease.
Investment, collaboration, and coordination have been key
Gemcitabine-associated thrombotic microangiopathy (TMA) is believed to be very rare, with an estimated incidence rate of 0.015%. Indications for gemcitabine are expanding, and comprehensive characterization this complication therefore important.The authors performed a retrospective chart review all cases gemcitabine-associated TMA diagnosed at Partners Healthcare System (Boston, MA) between January 1997 February 2002.Nine patients were identified. Diagnosis was aided by clinical laboratory...