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Cystic Fibrosis Weekly Update Contents: Screening Finds Fewer Newborns in Ireland With CF Than Expected ELX-02, Potential Therapy for CF Nonsense Mutations, Named Orphan Drug Screening Finds Fewer Newborns in Ireland With CF Than Expected Aug 07, 2020 07:00 am | Ines Martins, PhD A national newborn screening (NBS) program in the Republic of Ireland suggests that the incidence of cystic fibrosis and CF carriers in the country is lower than previously estimated, a study found. It also shows that the Gly551Asp mutation is almost three times more common among newborns in Ireland than in other parts of Europe and […] The post Screening Finds Fewer Newborns in Ireland With CF Than Expected appeared first on Cystic Fibrosis News Today. Read More ELX-02, Potential Therapy for CF Nonsense Mutations, Named Orphan Drug Aug 06, 2020 07:00 am | Marisa Wexler MS ELX-02, an investigational treatment of cystic fibrosis due to nonsense mutations, has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). This designation is given to treatments with the potential to improve care for rare diseases, defined as those affecting fewer than 200,000 people in the U.S. It qualifies its developer, Eloxx Pharmaceuticals, to benefits […] The post ELX-02, Potential Therapy for CF Nonsense Mutations, Named Orphan Drug appeared first on Cystic Fibrosis News Today. Read More Recent News Scottish Government and Vertex Strike Deal for Access to Kaftrio Putting Body Image Issues Behind Me: A #ScarPositive Story Higher Prevalence of Oral Candida Fungus Found in CF Patients An Open Letter to the Chronically Ill Professional Northern Ireland Reaches Deal With Vertex for Access to Kaftrio