Results 41 to 50 of about 1,802,515 (269)

Lyophilized monoclonal antibody (mAb) encoded mRNA—lipid nanoparticles (LNPs) lead to stable in vivo expression of mAb after room temperature storage

AAPS Open
The recent success of mRNA COVID-19 vaccines using lipid nanoparticles (LNPs) underscores the potential of this technology for delivering gene-encoded biologics in humans.
Zimeng Wang, Bei Cheng, Christine Tkaczyk, Michael Newton, Shuolin Cui, Bohdan Andreiuk, Sanjib Saha, Sabrina Khan, Charles Chen, Bin Yang, Adam Gamson, Sarah Siddiqui, George Thom, Antonio DiGiandomenico, Liping Zhou   +14 more
doaj   +1 more source

Dystrophic Epidermolysis Bullosa (DEB): How Can Pregnancy Alter the Course of This Rare Disease? An Updated Literature Review on Obstetrical Management with an Additional Italian Experience

Diseases
Epidermolysis Bullosa (EB) is an extremely rare and disabling inherited genetic skin disease with a predisposition to develop bullous lesions on the skin and inner mucous membranes, occurring after mild friction or trauma, or even spontaneously.
Antonella Vimercati, Gerardo Cazzato, Lucia Lospalluti, Stefania Foligno, Cristina Taliento, Katarzyna Beata Trojanowska, Ettore Cicinelli, Domenico Bonamonte, Dario Caliandro, Amerigo Vitagliano, Pierpaolo Nicolì   +10 more
doaj   +1 more source

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

The Case Study of Implementing the Delivery Optimization System at a Fast-Moving Consumer Goods Distributer

Promet (Zagreb), 2013
Using new optimization methods and information-communications technology has become the key issue in the competition among the distributers of fast-moving consumer goods.
Ante Galić, Tonči Carić, Juraj Fosin
doaj   +1 more source

Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection

Molecular Therapy: Nucleic Acids, 2023
Antisense oligonucleotide (ASO) therapeutics are being investigated for a broad range of neurological diseases. While ASOs have been effective in the clinic, improving productive ASO internalization into target cells remains a key area of focus in the ...
Amy E. Byrnes, Sara L. Dominguez, Chun-Wan Yen, Benjamin I. Laufer, Oded Foreman, Mike Reichelt, Han Lin, Meredith Sagolla, Kathy Hötzel, Hai Ngu, Christoffer Soendergaard, Alberto Estevez, Hsiu-Chao Lin, Alexandre Goyon, Juan Bian, Jessica Lin, Flora I. Hinz, Brad A. Friedman, Amy Easton, Casper C. Hoogenraad   +19 more
doaj   +1 more source

Ironic Medications: A Narrative‐Based Psycho‐Educational Intervention to Explore Patient–Provider Communication in Adolescents With Haematological Cancer

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Adolescents with haematological malignancies face significant emotional and relational challenges, often accompanied by difficulties in communicating their needs within the healthcare context. To address these issues, a narrative‐based psycho‐educational intervention based on the creation and prescription of Ironic Medications was ...
Marta Stoppa, Elena Pagani Bagliacca, Selena Russo, Giulia Rampoldi, Maria Grazia Strepparava, Adriana Cristina Balduzzi, Andrea Biondi, Marco Spinelli   +7 more
wiley   +1 more source

Re‐Irradiation in Pediatric Diffuse Midline Glioma: A Multi‐Institutional Retrospective Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Children with recurrent diffuse midline gliomas (DMGs) have limited therapeutic options at recurrence. Re‐irradiation (RT2) may be used at progression, but with uncertainty about the benefit. Methods We conducted a multi‐institutional retrospective study of children aged < 18 with DMG treated at three centers (Toronto, Canada ...
Ajay Thomas Alex, Sean P. Hassan, Helen Benghiat, Liam Herbert, Jennifer Adamski, Shivani Bailey, Claire Esler, Louise Brookes, Molly Bamber, Grace Koudou, Karen Waite, Julie Bennett, Anirban Das, Derek S. Tsang   +13 more
wiley   +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

BMT4me En Español: Multisite Feasibility and Usability Testing of a Spanish‐Language mHealth Adherence Support App for Spanish‐Speaking Caregivers of Children After Hematopoietic Stem Cell Transplantation and Cancer Treatment

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Medication nonadherence during the first 100 days after pediatric hematopoietic stem cell transplantation (HSCT) and during oncology treatment increases risk for complications. BMT4me is a caregiver‐facing mobile health (mHealth) application providing medication reminders, symptom tracking, and note‐taking features to support ...
Micah A. Skeens, Madeline R. Peek, Bianca Franklin, Emre Sezgin, Melissa Beauchemin   +4 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