Cystic fibrosis is caused by gene mutations that result in an abnormal Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein on the surface of cells. CFTR modulators are a novel class of drugs that directly target the molecular defect.…
November 2017
The Remaining Barriers to Normalcy in Cystic Fibrosis III
Powerful tools for genetic modification: Advances in gene editing
Recent discoveries and technical advances in genetic engineering, methods called gene or genome editing, provide hope for repairing genes that cause diseases like cystic fibrosis (CF) or otherwise altering a gene for therapeutic benefit. There are both hopes and hurdles with…
Mapping targetable inflammation and outcomes with cystic fibrosis biomarkers
Cystic fibrosis is characterized by an overly exuberant neutrophilic inflammatory response to pathogens and other stimuli that starts very early in disease. The overwhelming nature of this response is a primary cause of remodeling and destruction of the…
Nontuberculous mycobacteria in cystic fibrosis: Updates and the path forward
Nontuberculous mycobacteria (NTM) are troublesome pathogens that can cause significant pulmonary disease in patients with cystic fibrosis (CF). Diagnosis can be difficult in the setting of underlying CF and treatment regimens are burdensome on both patients and providers.…
CF‐related diabetes: Containing the metabolic miscreant of cystic fibrosis
Cystic fibrosis‐related diabetes (CFRD) is associated with both an increase in morbidity and mortality in people with cystic fibrosis (CF). With increased screening and improved life expectancy of people with CF, the prevalence of CFRD is expected to…
Data that empower: The success and promise of CF patient registries
In this article, we describe existing CF registries with a focus on US registry data collected through the CF Foundation Patient Registry (CFFPR) and the Epidemiologic Study of CF (ESCF); highlight what registries have taught us regarding epidemiology…
Overcoming barriers to a successful transition from pediatric to adult care
As life expectancy for people with cystic fibrosis (CF) has increased dramatically, so has the need for a guided, structured transition from pediatric to adult‐focused care. A formalized transition program allows for seamless transfer of patients between providers,…
Pediatric lung transplantation and end of life care in cystic fibrosis: Barriers and successful strategies
Pediatric lung transplantation has advanced over the years, providing a potential life‐prolonging therapy to patients with cystic fibrosis. Despite this, many challenges in lung transplantation remain and result in worse outcomes than other solid organ transplants. As CF…