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Starting Kalydeco Treatment Before Birth May Help Halt CF Progression Post-Birth, Animal Study Suggests

Treating an animal model of cystic fibrosis (CF) with Kalydeco (ivacaftor) during embryonic development and after birth revealed that early treatment improved pancreatic function, delayed lung disease, and reduced infections, a study suggests. The study, titled “In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model…

Synspira’s CF Therapy Candidate SNSP113 Granted Orphan Drug Status in EU

Synspira Therapeutics’s candidate for treating cystic fibrosis, SNSP113, has received orphan drug designation from the European Medicines’ Agency (EMA) in the European Union. The status was granted by the EMA’s Committee for Orphan Medicinal Products (COMP), which reviews experimental treatments developed for rare diseases. Thick mucus accumulates…

Phase 2a Trial Will Test Molgradex in CF Patients with NTM Infections

Savara Pharmaceuticals has launched a Phase 2a clinical study to evaluate the effectiveness of Molgradex in cystic fibrosis (CF) patients with chronic nontuberculous mycobacterial (NTM) lung infections. Molgradex is an inhaled form of artificially produced human protein granulocyte-macrophage colony-stimulating factor (GM-CSF), which normally is produced and secreted by…