Modern prophylactic therapies for hereditary angioedema with C1 inhibitor deficiency (HAE-C1INH) have become increasingly prevalent in Hungary, paralleling global trends, and are proving both ...

The US Food and Drug Administration (FDA) has granted a Priority Review designation to a New Drug Application (NDA) for TransCon ® CNP (navepegritide) for the treatment of children with achondroplasia ...

Physical activity before or during pregnancy was not associated with negative maternal/neonatal outcomes; however, physical activity was not statistically significantly associated with positive ...

Capillary zone electrophoresis appears to effectively identify molecular fragments capable of binding to human transthyretin (TTR), with potential therapeutic applications for transthyretin amyloid ...

A CRISPR/Cas9-modified Janus kinase 2 (JAK2) V617F model may be useful to develop new therapies against myeloproliferative neoplasms (MPNs), showed a new study published in the International Journal ...

The presence of Raynaud’s phenomenon and assessment with nailfold videocapillaroscopy (NVC) may identify patients with systemic lupus erythematosus (SLE) who are at high risk of pulmonary arterial ...

Venetoclax (VEN)-based therapies are effective after disease progression while receiving covalent Bruton tyrosine kinase inhibitors (cBTKi) in patients with chronic lymphocytic leukemia (CLL), ...

Stem cell therapy, artificial womb technology, and telehealth innovations are reshaping neonatal care and improving outcomes for infants affected by complex conditions, including fetal and neonatal ...

Researchers from China synthesized a new caffeic acid borate-derived linear polyglycidol polymer micelle (CAPG) to deliver a small interfering RNA (siRNA) to inflammatory sites in the liver that can ...

Patients with severe hemophilia A and B who develop inhibitors exhibit fundamentally distinct immune responses to replacement factor VIII and factor IX, as revealed by single-cell sequencing of ...

Researchers in the United States have observed an increase in the incidence of systemic mastocytosis (SM), particularly among non-Hispanic white individuals. SM is a rare mast cell disorder ...

Risk factors for thymidine kinase 2 deficiency (TK2d) include inheritance patterns, molecular pathogenesis, genotype-phenotype correlations, and the risk of secondary complications after disease onset ...