The FDA has granted Orphan Drug Designation to a novel inhaled treatment that could potentially improve lung function in patients with cystic fibrosis.
Forskolin-induced swelling in intestinal organoids is a clinically relevant biomarker of disease severity in infants diagnosed with cystic fibrosis.
Evidence is lacking that demonstrates that monotherapy with correctors has a clinically important effect on individuals with cystic fibrosis who have 2 copies of the F508del mutation.
First-phase and glucose-potentiation of arginine-induced insulin secretion as assessed by acute C-peptide responses improved after the initiation of ivacaftor in children with cystic fibrosis.
Lung transplantation can extend survival in patients with cystic fibrosis who have compromised lung function and increasing exacerbations.
The approval was supported by data from ARRIVAL (N=25), an ongoing Phase 3 open-label safety study in children with CF aged 12 to <24 months with 1 of 10 CFTR gene mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H).
In adult-diagnosed cystic fibrosis, diabetes, lung function, and age at diagnosis may predict patient survival.
The approval was based on data from a 24-week, open-label, Phase 3 study in patients aged 2 to 5 years.
A higher prevalence of anaerobic bacteria in cystic fibrosis was associated with higher lung function, less use of antibiotics, increased BMI, pancreatic sufficiency, and no requirement for insulin.
Compared with placebo, the risk for a pulmonary exacerbation was reduced by 44% in participants with cystic fibrosis who received azithromycin.
Ivacaftor decreased the risk for death, transplantation, hospitalization, and pulmonary exacerbation in patients with cystic fibrosis.
Sputum viscoelastic properties were associated with lung function and disease status in patients with cystic fibrosis.
Proteostasis Therapeutics is developing the combination treatment which includes a novel transmembrane conductance regulator (CFTR) amplifier (PTI-428), a third generation corrector (PTI-801) and a potentiator (PTI-808).
Patients with cystic fibrosis who took azithromycin had a significantly lower risk for detection of new nontuberculous mycobacteria, methicillin-resistant Staphylococcus aureus, and Burkholderia cepacia complex.
Sputum cultures could aid clinicians in choosing an appropriate antibiotic for patients with cystic fibrosis and avoiding allergies and antibiotic resistance.
A new cystic fibrosis transmembrane conductance regulator modulator treatment has been approved by the FDA for use in patients aged 12 years and older.
CFTR modulator therapy recommended in certain patients with cystic fibrosis.
Effective cystic fibrosis transmembrane conductance regulator modulator therapy may be of benefit in subsets of patients with cystic fibrosis.
In children with cystic fibrosis, risk for first detection of S aureus and P aeruginosa was greater in those who had received antibiotic prophylaxis.
Palliative care access may be limited for patients with cystic fibrosis.
The Adult ADHD Self-Report Scale-v1.1 Symptom Checklist may be able to detect previously undiagnosed ADHD in patients with cystic fibrosis.
Underlying cystic fibrosis may be a risk factor for the prediction of delayed gastric emptying in patients who undergo lung transplantation.
Lower microbial diversity in the respiratory tract in patients with cystic fibrosis was linked with prophylactic antibiotics and less airway inflammation.
Sweat chloride can predict severity in patients with CF, especially for long-term lung damage.
Combination lumacaftor/ivacaftor therapy was found to be safe and effective in patients with cystic fibrosis aged 6 to 11 years.
The FDA has expanded the approved indication of ivacaftor for cystic fibrosis, increasing the number of rare gene mutations the drug may be used to treat.
Inpatient antibiotic therapy is associated with greater likelihood of recovery following acute lung function decline in pediatric patients with cystic fibrosis.