Real-world romiplostim dosing, response and home administration in paediatric immune thrombocytopenia. [PDF]
Harris EM, Montcrieff C, Grace RF.
europepmc +1 more source
openaire +1 more source
Objective To characterize the clinical, immunologic, and proteomic changes associated with CD19 chimeric antigen receptor T cell therapy in patients with progressive systemic sclerosis (SSc). Methods Patients with progressive SSc received CD19 chimeric antigen receptor (CAR)‐T cell therapy and were observed longitudinally for safety, clinical efficacy,
Chenhan Jia +16 more
wiley +1 more source
Pregnancy outcomes among women with inherited bleeding disorders: A matched case-control study. [PDF]
Mansouri Z +10 more
europepmc +1 more source
Human Monocytic Models Reveal Genotype‐Dependent Inflammatory Programs in VEXAS Syndrome
Objectives VEXAS syndrome is a severe X‐linked autoinflammatory disorder caused by somatic mutations in ubiquitin‐like modifier activating enzyme 1 (UBA1), with clinical outcomes that vary by UBA1 genotype. We aimed to elucidate genotype‐specific inflammatory programs and identify potential therapeutic targets.
Kana Higashitani +10 more
wiley +1 more source
The Association Between Peripheral Complete Blood Count Parameters and Depressive Symptoms: A Cross-Sectional Study in Eastern Sudan. [PDF]
Alharthi SS +4 more
europepmc +1 more source
Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis +4 more
wiley +1 more source
Late Vitamin K Deficiency Bleeding Presenting as Subacute Subdural Hemorrhage in an Exclusively Breastfed Infant: A Preventable Life-Threatening Condition-A Case Report. [PDF]
Ali YK +7 more
europepmc +1 more source
Aim The purpose of this study is to evaluate safety, tolerability, pharmacokinetics (PK), food‐effect (FE) and pharmacodynamics (PD) of an oral tyrosine kinase‐2 (TYK2)/Janus kinase‐1 (JAK1) inhibitor, SDC‐1801, in healthy adult participants. Methods This first‐in‐human study randomized 95 male and female participants.
Chris Brearley +3 more
wiley +1 more source
Eltrombopag use in surgical procedures in inherited thrombocytopenia: expanding the role of thrombopoietin-receptor agonists. [PDF]
Oliveira M +3 more
europepmc +1 more source

