The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible
progress in treating the disease such that the mean survival age of individuals living with CF …

Abstract Purpose of Review Chronic rhinosinusitis with nasal polyps (CRSwNP) is a highly
prevalent disease that results in significant healthcare-related costs as well as costs to …

Objectives Lumacaftor–ivacaftor is a cystic fibrosis transmembrane conductance regulator
(CFTR) modulator known to improve clinical status in people with cystic fibrosis (CF). The …

ABSTRACT Introduction Cystic Fibrosis (CF) is caused by mutations in the Cystic Fibrosis
Transmembrane Conductance Regulator (CFTR) gene. The most common mutation …

Abstract Background and Objectives Rare diseases have a significant impact on patients,
families, the health system, and society. Measuring the socioeconomic burden is crucial to …

Preterm birth remains one of the most urgent unresolved medical problems in obstetrics, yet
only 2 therapeutics for preventing preterm birth have ever been approved by the United …

Cystic Fibrosis (CF) is the most common, severe, autosomal recessive disease in the
Caucasian population, and is caused by mutations in the cystic fibrosis transmembrane …

BACKGROUND: Cystic fibrosis (CF) is the most serious and frequent hereditary autosomal
disease that causes respiratory, hepatic and pancreatic dysfunction. The aim of the study …

Background: Cystic fibrosis (CF) is the most serious and frequent hereditary autosomal
disease that causes respiratory, hepatic and pancreatic dysfunction. The aim of the study …

O debate sobre a introdução, a utilização e o investimento em tecnologias de saúde
inovadoras repete-se em todos os sistemas de saúde. As tecnologias que se apresentam …