Developing a first-in-class regenerative gene therapy for cystic fibrosis
OmniSpirant Limited is an Irish company developing a regenerative gene therapy with the potential to transform respiratory medicine. The...
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Toddlers Age 1-2 With CF Can Now Be Treated With Kalydeco in Canada
Health Canada has approved the use of Kalydeco (ivacaftor) in toddlers age 1-2 years with certain genetic mutations that prevent the...
FDA puts Translate Bio’s rare disease mRNA drug on hold
The FDA has placed a clinical hold on the IND for Translate Bio’s mRNA treatment for ornithine transcarbamylase (OTC) deficiency, a...
The Diverse Spectrum of Health Across the CF Community
If you’ve ever met more than one person with cystic fibrosis, it’s likely they had very different experiences than you. First, their...
Non-invasive Ventilation Improves Lung Function in CF Patients But Not Survival, Study Finds
A reassessment of U.K. medical records showed that in cystic fibrosis (CF) patients, breathing support provided by gas inhalation,...
Eluforsen Improves CFTR Protein Function in CF Patients with F508del Mutations, Phase 1 Trial Shows
An exploratory Phase 1 trial showed that repeated intranasal treatment with the therapy eluforsen improved CFTR protein activity in...
Aridis Enrolling CF Patients to Test AR-501 in Chronic Lung Infections
Aridis Pharmaceuticals has enrolled the first healthy participant in its Phase 1/2a clinical trial to evaluate the antibacterial...
Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests
The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically...
