Abstract While lung disease continues to be the main cause of morbidity and mortality, the improved survival rates have meant that nonpulmonary complications have become increasingly important in the care…
Abstract This chapter describes the therapeutic strategies for cystic fibrosis which are based on targeting cystic fibrosis transmembrane conductance regulator (CFTR), either at the gene or protein level. We provide…
Abstract Pulmonary thromboembolism (PTE) is an uncommon but potentially life-threatening condition in pediatrics. Most children with venous thromboembolism (VTE) and PTE share common risk factors that include vascular endothelial injury…
Abstract Cystic fibrosis (CF) is the most common, life-shortening inherited disease of Caucasians. Much of its morbidity and mortality is related to progressive airway involvement, the consequence of defective function…
Abstract Pulmonary problems in children with sickle cell disease (SCD) cause significant morbidity and mortality. Acute chest syndrome (ACS) is the leading cause of death, and recurrent ACS episodes are…
Abstract Congenital heart disease affects nearly 1% of births. Unrepaired cardiac disease, the postoperative state after congenital heart surgery, and long term of sequelae of certain congenital repairs can significantly…
Abstract This chapter characterizes selected disorders with a primary respiratory component as well as respiratory manifestations of systemic diseases. Respiratory disorders of the lymphatic system may present at any age…
Abstract Our understanding of the role of disease-causing variants in CFTR has culminated in two new variant-specific CFTR modulator therapies approved by the US FDA. These modulators target defects in…
Abstract Most children with cystic fibrosis (CF) in current times are likely to be picked up through newborn screening programs and confirmed by the finding of two CF-causing mutations and…
Abstract Asthma occurs as a result of the interplay of genetic susceptibility and environmental influences on the developing lungs and immune system. Asthma in children is predominantly associated with the…
