The development of safe and efficacious enzyme-based human therapies has increased
greatly in the last decades, thanks to remarkable advances in the understanding of the …

The growing population of adults with cystic fibrosis (CF) is not a chance occurrence. This
exemplar of medical innovation and clinical success is the product of engaged, informed …

This study provides detailed data on the current characteristics, perceptions and outcomes
of 45 young people with cystic fibrosis (CF) as they transition into adulthood. Although many …

Cystic fibrosis (CF) is an autosomal recessive, inherited congenital disease caused by the
mutation of the family autosomal CF gene, with cumulative exocrine secretion characterized …

Background Improved survival has led to increasing numbers of children with life-limiting
conditions transitioning to adult healthcare services. There are concerns that transition may …

Survivors of adolescent and young adult (AYA) cancers, defined as individuals diagnosed
with a primary malignancy between age 15 and 39 years, are a growing population with …

Skin fibroblasts obtained from a 27-year-old man with clinically manifested and genetically
proven (F508del/F508del) cystic fibrosis were successfully transformed into induced …

Skin fibroblasts obtained from a 28-year-old man with clinically manifested and genetically
proven (F508del/W1282X) cystic fibrosis were successfully transformed into induced …

Objective Previous research exploring patient-practitioner communication in relation to
adherence in cystic fibrosis (CF) is limited. This UK study explored the views of adults with …

Cystic fibrosis is one of the most common inherited diseases caused by mutations in CFTR
gene, of which F508del is the most frequent. Currently, the possibility of cell therapy …