Results 51 to 60 of about 2,774 (200)

Efficacy and safety of empagliflozin for treating neutropenia and neutrophil dysfunction in paediatric patients with glycogen storage disease type Ib: A systematic review and meta‐analysis

British Journal of Clinical Pharmacology, EarlyView.
Aims Glycogen storage disease type Ib (GSD‐Ib) is a rare genetic disorder causing neutropenia and neutrophil dysfunction in children. G‐CSF has been the primary treatment, but emerging data support the potential of empagliflozin, an SGLT2 inhibitor, as a promising investigational option.
Elizabeth Iwasyk, Ryan Jin, Fabio Tuzzolino, Giusy Ranucci, Donatella Madonia, Alessio Provenzani   +5 more
wiley   +1 more source

First‐in‐human, phase I, randomized, safety, pharmacokinetic, food‐effect and pharmacodynamic study of a tyrosine kinase 2/Janus kinase 1 inhibitor, SDC‐1801

British Journal of Clinical Pharmacology, EarlyView.
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, Sam Francis, John Reader, Judi Proctor   +3 more
wiley   +1 more source

Targeting neutrophil extracellular traps in metabolic and immune niche: Nanomaterials for diabetes tissue regeneration

BMEMat, EarlyView.
The effects of NETs on regeneration of various diabetic tissues, and strategies targeting NETs for diabetes tissue regeneration. In the diabetic environment, NETs undergo complex metabolic and immune reprogramming, leading to dynamic changes in antibacterial and proinflammatory functions, and affecting regeneration of multiple systemic tissues.
Xinyi Jiang, Muxin Yue, Kai Mao, Boon Chin Heng, Noor Azlin Yahya, Yunsong Liu, Zheng Li   +6 more
wiley   +1 more source

Nanocubes and Cuboctahedra: Entropic Stabilization Versus Thermal Segregation in Rock Salt‐ and Spinel‐Type Multinary Oxides from Spray‐Flame Synthesis

ENERGY &ENVIRONMENTAL MATERIALS, EarlyView.
Spray‐flame synthesis enables highly flexible and scalable production of multinary and high‐entropy oxide nanoparticles due to high synthesis temperatures with subsequent quenching. Temperature‐dependent in situ diffraction of as‐synthesized materials clearly reveals entropy stabilization in rock salt lattices, while spinels—featuring tetrahedral and ...
Mohammed‐Ali Sheikh, Steven Angel, Sabrina Schleich, Leon Müller, Jan Ternieden, Hatem M. A. Amin, Christof Schulz, Hartmut Wiggers   +7 more
wiley   +1 more source

A novel neuromuscular form of glycogen storage disease type IV with arthrogryposis, spinal stiffness and rare polyglucosan bodies in muscle

, 2016
Glycogen storage disease type IV (GSD IV) is an autosomal recessive disorder causing polyglucosan storage in various tissues. Neuromuscular forms present with fetal akinesia deformation sequence, lethal myopathy, or mild hypotonia and weakness.
Gitiaux, C., Barnerias, C., Oldfors, Anders,, Carlier, R. Y., Romero, N. B., Oldfors Hedberg, Carola,, Benezit, A., Quijano-Roy, S., Malfatti, E.   +8 more
core   +1 more source

Geochemistry and Geochronology of Magmatic Rocks in Southwestern and Northeastern Cambodia: Implications for the Tectonic Evolution and Metallogenesis of the Loei Fold Belt

Geological Journal, EarlyView.
The composition of intrusive rocks suggests that the magma was likely generated in a subduction‐related setting. The arc crustal thickness in the Phnom Sro Ngam and Halo Prospects was probably < 40 km during emplacement. Zircon U–Pb age range indicates a correlation with Loei Fold Belt magmatic activity.
Sirisokha Seang, Khin Zaw, Kakda Kret, Ivan Belousov, Kotaro Yonezu, Koichiro Watanabe, Charles Makoundi   +6 more
wiley   +1 more source

Null mutations and lethal congenital form of glycogen storage disease type IV.

, 2007
Glycogen branching enzyme deficiency (glycogen storage disease type IV, GSD-IV) is a rare autosomal recessive disorder of the glycogen synthesis with high mortality.
Diogo, L, Henriques, M, Rebelo, O, Diggelen, O.P. van, Boode, W.P. de, Dilling, J, Carreira, I., Rebelo, O., Carreira, I, Bruno, C., Garcia, P, Assereto, S., Assereto, S, Morava, E., ter Laak, H, Cassandrini, D, Morava, E, Laak, H.J. ter, Minetti, C., Cassandrini, D., de Boode, WP, Garcia, P., Dilling, J., Henriques, M., Bruno, C, Diggelen, Otto, Diogo, L., Minetti, C   +27 more
core   +1 more source

Sono‐Vasomics With Super‐Resolution Contrast‐Enhanced Ultrasound (SR‐CEUS) for Characterization of Focal Liver Lesions: A Case Report

Portal Hypertension &Cirrhosis, EarlyView.
ABSTRACT Accurate differentiation between benign and malignant focal liver lesions (FLLs) remains a major diagnostic challenge. Vasomics, an emerging discipline, classifies vascular phenotypes into anatomical, biomechanical, biochemical, pathophysiological, and composite categories. Sono‐Vasomics, the ultrasound‐based subset, leverages super‐resolution
Yijie Qiu, Ying Wang, Rui Cheng, Ruiling He, Yi Dong, Christoph F. Dietrich   +5 more
wiley   +1 more source

Continuous glucose monitoring metrics in people with liver glycogen storage disease and idiopathic ketotic hypoglycemia: A single-center, retrospective, observational study [PDF]

Background: Cohort data on continuous glucose monitoring (CGM) metrics are scarce for liver glycogen storage diseases (GSDs) and idiopathic ketotic hypoglycemia (IKH).
Schreuder A. B., Venema A., Overduin R. J., Lubout C. M. A., Rossi A., Fokkert-Wilts M. J., Derks T. G. J., De Boer F.   +7 more
core   +2 more sources

Evaluation of glycogen storage patients: Report of twelve novel variants and new clinical findings in a Turkish population

, 2021
© 2021 by the authors. Licensee MDPI, Basel, Switzerland.Glycogen storage diseases (GSDs) are clinically and genetically heterogeneous disorders that disturb glycogen synthesis or utilization.
GEDİKBAŞI, Asuman, Bulent Uyanik, Asuman Gedikbasi, Uyanik, Bulent, Ersoy, Melike, Melike Ersoy   +5 more
core   +1 more source