News

Proteostasis’s CF Treatment Obtains Breakthrough Therapy Designation from FDA

The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to Proteostasis Therapeutics’ cystic fibrosis therapy PTI-428 — a move aimed at accelerating its development. Mutations of the CFTR gene cause CF. The most common is F508del, which leads to faulty CFTR protein. Normal CFTR protein is required for transporting chloride ions…

Diverse Types of Staphylococcus Aureus Found in Airways of CF Patients, Study Shows

Dozens of diverse types of the bacteria Staphylococcus aureus were identified in the airways of cystic fibrosis (CF) patients, including five new types and one commonly associated with livestock, a study reports. Findings were reported in the study, “Emergence and spread of worldwide Staphylococcus aureus clones among cystic fibrosis patients, published in the…

Ibuprofen Counters Key Bacteria in Lungs of CF Patients, Study Shows

Ibuprofen does a good job of fighting bacteria in cystic fibrosis (CF) patients’ lungs, including two key threats, Pseudomonas aeruginosa and Burkholderia, a study shows. Researchers said the findings support ibuprofen’s use as an add-on therapy against bacterial infections in CF. The study, “Antimicrobial Activity of Ibuprofen against Cystic Fibrosis-Associated Gram-Negative…

Two Bone Density Scanning Methods Show Similar Results in CF Children

Prepubescent children with cystic fibrosis (CF) have lower bone density compared to healthy children, and findings are comparable using two distinct bone density scanning methods, researchers report. The study that supports that conclusion, “Peripheral quantitative computed tomography detects differences at the radius in prepubertal children with cystic…

Report Calls Attention to Burden of Rare Disease Caregivers

Caregivers of people with rare diseases face emotional, financial, physical, and social strain and receive little outside support, a U.S. study found. The findings will be presented to Congress this spring along with policy recommendations to help support those caring for people with rare diseases. The report is titled “…