cystic fibrosis

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…

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…

Eloxx Pharmaceuticals presented new positive preclinical data for ELX-02, its lead treatment candidate for cystic fibrosis (CF), demonstrating that the therapy increases the levels of CFTR protein and its correct placement at the cell’s surface in patient-derived organoids. Findings were presented by Shira Landskroner-Eiger, PhD, Eloxx’s principal…

Your CF Community


Visit the Cystic Fibrosis News Today forums to connect with others in the CF community.