Results 41 to 50 of about 1,336,426 (267)

Personalized Zebrafish Models for Fusion‐Positive Pediatric Sarcomas

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Clinical sequencing efforts have revolutionized our approaches to categorizing pediatric cancers in real time. This has dramatically improved our ability to profile pediatric tumors, identify actionable vulnerabilities, and influence clinical care.
Lisa H. Hall, Emma N. Harrison, Genevieve C. Kendall   +2 more
wiley   +1 more source

Incidence and prevalence of light chain amyloidosis in the United States in 2019–2021 using Optum EHR data

Scientific Reports
Immunoglobulin light chain amyloidosis (AL amyloidosis) is among the most common forms of systemic amyloidosis. Using electronic health records (EHR) data from the United States, we aimed to estimate the incidence and prevalence of AL amyloidosis over ...
Pedro A. Laires, Shona Fang, Jennifer Evans, Jeffrey Thompson, Abhishek Gaur, Brendon Staruk, Ankita Gupta, Richa Manwani   +7 more
doaj   +1 more source

Whipple's disease with constrictive pericarditis: A rare disease with a rare presentation [PDF]

Canadian Journal of Cardiology, 2009
Whipple's disease is a multisystem disease that can affect the heart with predominantly endocardial and pericardial involvement and, less often, myocardial inflammation. Previously diagnosed at autopsy, cardiac involvement in Whipple's disease is being recognized clinically more often. A 58-year-old man with Whipple's-related constrictive pericarditis,
T, Iqbal, A, Karovitch, J, Veinot, R, Saginur, L, Beauchesne   +4 more
openaire   +2 more sources

The Role of “Adult‐Onset” Cancer Predisposition Genes in Pediatric Cancer: A Comprehensive Review

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Current literature estimates that 10% of pediatric cancers are caused by pathogenic or likely pathogenic (P/LP) germline variants in cancer predisposition genes (CPGs). Variants in CPGs thought to increase cancer risk exclusively during adulthood are referred to as “adult‐onset” CPGs (aoCPGs).
Maria Rozo, Miriam A. Bornhorst, Angela J. Waanders, Sara Zarnegar‐Lumley, Ashwin Lakshman Koppayi, Lucy A. Godley   +5 more
wiley   +1 more source

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of ALXN1820 (Tarperprumig) in Healthy Adults: Results of a Phase I Study

Clinical and Translational Science
Properdin is an endogenous positive regulator of the complement alternative pathway (AP). Tarperprumig (ALXN1820), a novel humanized bispecific antibody, binds properdin and albumin and is being developed to treat complement‐mediated diseases. This phase
Avner Sandhu, Tong Shen, Paz Martin Herrero, Chao‐Xing Yuan, Samirah Qureshi, Xiaoyu Jiang, Ye Sheng, Christoph Gasteyger, Yang Dai   +8 more
doaj   +1 more source

Association Between Dinutuximab Beta Exposure and Post‐End‐of‐Treatment Survival in Neuroblastoma: A Weighted Patient‐Level Analysis of Three Clinical Studies

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Objectives The association between exposure to dinutuximab beta (DB) and event‐free survival (EFS) or overall survival (OS) of neuroblastoma patients was assessed using data collected during three clinical trials (five cohorts). Methods A systematic review (March 2026) was conducted to identify relevant studies (prospective; registered DB ...
Przemysław Holko, Holger N. Lode, Alberto Garaventa, Carla Manzitti, Roberto Luksch, Dominique Valteau‐Couanet, Marion Gambart, John Anderson, Juliet Gray, Adela Canete, Lucas Moreno, Aleksandra Wieczorek, Cormac Owens, Shifra Ash, Isaac Yaniv, Genevieve Laureys, Hans Loibner, Katarzyna Śladowska, Ruth Ladenstein, Paweł Kawalec   +19 more
wiley   +1 more source

Summary of Research: Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis

Neurology and Therapy, 2023
This article provides a summary of a previously published paper: Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. The paper reported the results of the CHAMPION-MG trial which investigated the drug ravulizumab in the rare ...
Tuan Vu, Andreas Meisel, Renato Mantegazza, Djillali Annane, Masahisa Katsuno, Rasha Aguzzi, Ahmed Enayetallah, Kathleen N. Beasley, Nishi Rampal, James F. Howard   +9 more
doaj   +1 more source

T2* Magnetic Resonance Imaging Uncovers Hemosiderin Burden in Pediatric Hemophilia: A Call for Sensitive Imaging Biomarkers

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Hemophilic arthropathy remains the leading morbidity in hemophilia despite modern prophylaxis, and early joint damage may be missed by routine exams. This study explored T2* MRI as a noninvasive biomarker of hemosiderin deposition in pediatric hemophilia.
Jessica Garcia, Tarique Hussain, Jeanne Dillenbeck, Matthew Hammer, Annette von Drygalski, Guy Young, Ayesha Zia   +6 more
wiley   +1 more source

Survival by Race and Ethnicity in Children and Adolescents/Young Adults With Relapsed/Refractory Hodgkin Lymphoma: A Pooled Analysis of Children's Oncology Group Trials

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Purpose Despite 5‐year survival rates of over 90% among children and adolescents/young adults (CAYAs) with classic Hodgkin lymphoma (cHL), 15%–20% relapse after frontline therapy. Prior analysis of frontline Children's Oncology Group (COG) clinical trials demonstrated that, despite similar rates of relapse, non‐Hispanic Black (NHB) and ...
Mallorie B. Heneghan, Lindsay A. Renfro, Yue Wu, Katia M. Crisler, Erin Marcotte, Tanya Trippett, Terzah Horton, Bradford S. Hoppe, Frank G. Keller, Chris Forlenza, Cindy Schwartz, Kara M. Kelly, Peter D. Cole, Sharon M. Castellino, Justine M. Kahn   +14 more
wiley   +1 more source

Switching from Rituximab to Eculizumab in US Patients with Neuromyelitis Optica Spectrum Disorder: Impact on Hospitalizations

Neurology and Therapy
Introduction Neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease, affects the optic nerves and spinal cord. Rituximab is often used off-label to treat NMOSD; however, comparator-controlled trial data are limited.
Justin Lee, Adrian Kielhorn, Sami Fam, Emily Riser, Eoin P. Flanagan   +4 more
doaj   +1 more source