When North Americans say “supporting children’s lives and futures,” they mean well-being, opportunities, and health. In Ukraine, we are now talking about physical  survival, and we often narrow the meaning of the future to survive until tomorrow. “Children are its most vulnerable hostages,” said the First Lady of Ukraine, summari
zing the gap between peacetime expectations and our daily struggle for survival.

UDC 616-053.2:616.89-056.7:577.21

Neurofibromatosis type 1 is a common autosomal-dominant neurocutaneous disorder with broad molecular and clinical heterogeneity. Pediatric patients frequently present early with pigmentary findings, neurodevelopmental issues, and risk of tumor development, including plexiform neurofibromas. In this paper, we aimed to synthesize recent advances in the molecular genetics of neurofibromatosis type 1 and genotype-phenotype correlations, and to illustrate clinical variability through three anonymized pediatric cases.

The WIL-31 study demonstrated efficacy and safety of prophylaxis with the plasma-derived von Willebrand factor/factor VIII concentrate wilate in von Willebrand disease (VWD) of all types and was the only prospective study with an on-demand run-in study as an intra-individual comparator. This subgroup analysis examines the efficacy of wilate prophylaxis inpatients with type 3 VWD in WIL-31.