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 +5 more
wiley +1 more source
Surgical Colonic Myeloid Sarcoma Following Bone Marrow Transplantation in a Patient With Acute Myeloid Leukemia With Myelodysplasia-Related Changes: A Case Report. [PDF]
Ichikawa T +6 more
europepmc +1 more source
Precision medicine in paediatrics: Progress and priorities
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source
A Pilot Study of Bone Marrow Transplantation in a GALT-Null Rat Model of Classic Galactosemia. [PDF]
Rasmussen SA +6 more
europepmc +1 more source
Metabolomic investigation of cisplatin‐induced acute kidney injury in paediatric cancer patients
Aim Cisplatin causes acute kidney injury (AKI) in approximately 46% of paediatric cancer patients who receive it. Serum creatinine (SCr) is a poor biomarker of cisplatin nephrotoxicity, because there is a delay between cisplatin infusion and SCr elevation.
Yong Jin Lim +37 more
wiley +1 more source
AI-driven prognostics in pediatric bone marrow transplantation: a CAD approach with Bayesian and PSO optimization. [PDF]
Badawy M +7 more
europepmc +1 more source
Affinity Peptides With pH Sensitivity for the Enrichment of CD38+ Cells
ABSTRACT The selective enrichment of cell populations based on surface markers is critical for the advancement of gene and cell therapies. Current antibody‐based cell isolation methods, such as fluorescence‐ and magnetic‐activated cell sorting (FACS and MACS), offer high specificity but are limited by scalability, cost, and potential adverse effects on
Gabrielle Rusch +7 more
wiley +1 more source
Association of Donor and Recipient Duffy and Kidd Genotypes with GVHD in Leukemia Patients Undergoing Bone Marrow Transplantation. [PDF]
Mohammad HN +5 more
europepmc +1 more source

