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...