ABSTRACT Iptacopan is a first‐in‐class, oral, selective inhibitor of complement factor B that has demonstrated positive efficacy across several complement‐driven diseases. Here we evaluate the efficacy and safety of iptacopan monotherapy in adult patients with primary immune thrombocytopenia (ITP) and primary cold agglutinin disease (CAD). We performed
Alexander Röth +13 more
wiley +1 more source
Alterations in plasma cytokine profiles in generalized myasthenia gravis following different immunotherapeutic regimens. [PDF]
Ren Z +6 more
europepmc +1 more source
Essential role of caldesmon in the actin filament reorganization induced by glucocorticoids. [PDF]
Flora Castellino +3 more
openalex +1 more source
Identification of Compound Heterozygous CYP11A1 Variants via Reanalysis of Clinical Sequencing Data
ABSTRACT A molecular diagnosis is currently achievable in approximately 50% of patients assessed by clinical geneticists at tertiary care centres. Next‐Generation Sequencing Panels contain a defined group of genes associated with a clinically defined set of phenotypes.
Ana Acosta Bedón +10 more
wiley +1 more source
Impact of Psychological Stress-Derived Hormones and Cytokines on Immune Cell Profiles in Vitiligo: Toward Improved Peripheral Blood Simulation in Animal Models. [PDF]
Tang N +5 more
europepmc +1 more source
ABSTRACT Biallelic pathogenic variants in AKR1D1 cause Δ4‐3‐oxosteroid 5β‐reductase deficiency, disrupt bile acid synthesis, and result in Congenital Bile Acid Synthesis defect type 2 (CBAS2). CBAS2 presents in infancy with cholestasis, coagulopathy, and failure to thrive.
Jade Hudson +3 more
wiley +1 more source
Co-Occurrence of Endogenous and Exogenous Cushing's Syndromes: Does "Double Cushing Syndrome" Really Exist? A Case Report. [PDF]
Amani-Beni R +4 more
europepmc +1 more source
Dysregulation of Glucocorticoid Receptor Homeostasis and Glucocorticoid-Associated Genes in Umbilical Cord Endothelial Cells of Diet-Induced Obese Pregnant Sheep [PDF]
Eugenia Mata‐Greenwood +6 more
openalex +1 more source
ABSTRACT Background Nasal airway obstruction (NAO) is prevalent with substantial health and quality of life burdens. Nasal valve collapse (NVC) is one structural cause of NAO. Temperature‐controlled radiofrequency (TCRF) nasal valve remodeling offers an alternative to invasive surgery.
David W. Kennedy +7 more
wiley +1 more source

