Results 71 to 80 of about 18,093 (282)

Hematopoietic Stem Cell Transplantation in an Infant with Immunodeficiency, Centromeric Instability, and Facial Anomaly Syndrome

Frontiers in Immunology, 2017
Immunodeficiency, centromeric instability, and facial anomaly (ICF) syndrome is a rare autosomal recessive genetic condition with severe immunodeficiency, which leads to lethal infections if not recognized and treated in early childhood.
Katharina L. Gössling, Cyrill Schipp, Ute Fischer, Florian Babor, Gerhard Koch, Friedhelm R. Schuster, Jutta Dietzel-Dahmen, Dagmar Wieczorek, Arndt Borkhardt, Roland Meisel, Michaela Kuhlen   +10 more
doaj   +1 more source

Clinical case of primary immunodeficiency: X-linked agammaglobulinemia

Медицинская иммунология, 2020
Aclinical case ofprimary immunodeficiency state (PIDS) is described: X-linked agammaglobulinemia in the boy of 8 years old. The results of molecular genetic studies: gene btk (ex 1-19) genome version GRCh38.p5, transcript version ENST00000308731 single ...
E. V. Kuvschinova, L. M. Kurtasova, A. G. Borisov, A. R. Schmidt, T. A. Gerasimova   +4 more
doaj   +1 more source

X-Linked Agammaglobulinemia and COVID-19: Two Case Reports and Review of Literature

Pediatric Allergy, Immunology, and Pulmonology, 2020
Introduction: The Centers for Disease Control and Prevention (CDC) has listed primary immunodeficiency disorders as being predisposed to severe coronavirus disease 2019 (COVID-19).
F. M. Devassikutty, Abhinav R Jain, Athulya Edavazhippurath, Michael Chittettu Joseph, Mohammed Manekkat Thekke Peedikayil, V. Scaria, P. Sandhya, G. Govindaraj   +7 more
semanticscholar   +1 more source

Six‐Year Trends in Real‐World Data Use for Post‐Marketing Surveillance of New Medical Products in Japan

Clinical and Translational Science, Volume 19, Issue 2, February 2026.
ABSTRACT The Ministerial Ordinance on Good Post‐Marketing Study Practice for Drugs was amended by the Ministry of Health, Labour and Welfare (MHLW) in 2018 to clearly define post‐marketing database studies (DBS) as a measure of pharmacovigilance activities for approved medical products in Japan.
Suguru Okami, Kako Shimotsumagari, Yasunari Sadatsuki   +2 more
wiley   +1 more source

Understanding secondary hypogammaglobulinemia and its implications for cancer prognosis in children: A retrospective cohort study

Biomédica: revista del Instituto Nacional de Salud
Introduction. Immunodeficiencies are disturbances in the immune system that can affect cell function, quantity, or both. They can be either primary, associated with genetic defects, or secondary, linked to external factors such as hemato-oncological ...
Ana Lucía Guzmán, Isabella Villamil, Sofía Martínez-Betancur, Oriana Arias-Valderrama, Jacobo Triviño-Arias, Jessica Largo, Viviana Lotero, Alexis Franco, Ximena Castro, Pamela Rodríguez, Luz Angela Urcuqui, Diego Medina, Manuela Olaya   +12 more
doaj   +1 more source

Durability of Response to B‐Cell Maturation Antigen‐Directed mRNA Cell Therapy in Myasthenia Gravis

Annals of Clinical and Translational Neurology, Volume 12, Issue 11, Page 2358-2366, November 2025.
ABSTRACT Objective We report the 12‐month follow‐up outcomes from a Phase 2 clinical trial (NCT04146051) evaluating Descartes‐08, a BCMA‐directed RNA chimeric antigen receptor T‐cell (rCAR‐T) therapy for refractory generalized myasthenia gravis (MG).
Nizar Chahin, Gregory Sahagian, Marc H. Feinberg, C. Andrew Stewart, Christopher M. Jewell, Metin Kurtoglu, Miloš D. Miljković, Tuan Vu, Tahseen Mozaffar, James F. Howard Jr, the MG‐001 Study Group   +10 more
wiley   +1 more source

Identification of a novel BTK variant in a Chinese family with X-linked agammaglobulinemia [PDF]

Jichu yixue yu linchuang, 2021
Objective To identify the pathogenic variant in a Chinese family with X-linked agammaglobulinemia (XLA). Methods A trio family with suspected X-linked agammaglobulinemia was recruited.
ZHANG Han, SUN Yang, WANG Rong-rong, ZHANG Wen, ZHANG Xue
doaj  

Chapter Three. EED library as a basis for systematic reviews [PDF]

, 2016
3.1 Defining Systematic Review Question Priorities 3.2 Determining Relevance to the Systematic Review 3.3 Acquisition of References and Copyright Fair Use Compliance 3.4 Documenting Relevance to the Systematic Review 3.5 Data Extraction for the ...
Denno, Donna M, Musser, Christine A, Nelson, Zakia C, Tarr, Phillip I, VanBuskirk, Kelley M   +4 more
core   +1 more source

Case report: Rapidly progressive neurocognitive disorder with a fatal outcome in a patient with PU.1 mutated agammaglobulinemia

Frontiers in Immunology
IntroductionPU.1-mutated agammaglobulinemia (PU.MA) represents a recently described autosomal-dominant form of agammaglobulinemia caused by mutation of the SPI1 gene.
Rada Miskovic, Rada Miskovic, Jelena Ljubicic, Jelena Ljubicic, Branka Bonaci-Nikolic, Branka Bonaci-Nikolic, Ana Petkovic, Vladana Markovic, Vladana Markovic, Ivan Rankovic, Jelena Djordjevic, Jelena Djordjevic, Ana Stankovic, Kristel Klaassen, Sonja Pavlovic, Maja Stojanovic, Maja Stojanovic   +16 more
doaj   +1 more source

Ibrutinib inhibits SDF1/CXCR4 mediated migration in AML [PDF]

, 2014
Pharmacological targeting of BTK using ibrutinib has recently shown encouraging clinical activity in a range of lymphoid malignancies. Recently we reported that ibrutinib inhibits human acute myeloid leukemia (AML) blast proliferation and leukemic cell ...
Bowles, Kristian M, Lawes, Matthew J, MacEwan, David J, Murray, Megan Y, Rushworth, Stuart A, Shafat, Manar S., Zaitseva, Lyubov   +6 more
core   +2 more sources