Cystic Fibrosis News Today Weekly Digest

Cystic Fibrosis Weekly Update Contents: Let’s Use the Cystic Fibrosis News Today Forums for Empowerment Eluforsen Improves CFTR Protein Function in CF Patients with F508del Mutations, Phase 1 Trial Shows WHO Approves Ensifentrine as Name for Potential Inhaled Treatment Now in Clinical Trials New Inhaled Carrier May Work for CF mRNA Therapy, Mouse Study Finds Let’s Use the Cystic Fibrosis News Today Forums for Empowerment Jan 14, 2019 09:00 am | Tré LaRosa I ecstatically joined Cystic Fibrosis News Today in September because it was my first major opportunity to generate CF discussion. In this column, I’ve written about what a cure might actually mean for the community, why it’s important for parents to empower their children with CF, the bittersweet downside of modulators, exciting revelations from the […] The post Let’s Use the Cystic Fibrosis News Today Forums for Empowerment appeared first on Cystic Fibrosis News Today. Read More Eluforsen Improves CFTR Protein Function in CF Patients with F508del Mutations, Phase 1 Trial Shows Jan 14, 2019 07:00 am | Jose Marques Lopes, PhD An exploratory Phase 1 trial showed that repeated intranasal treatment with the therapy eluforsen improved CFTR protein activity in adults with cystic fibrosis (CF) carrying F508del mutations in both CFTR gene copies. The study, “Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis,” was published in the Journal of Cystic Fibrosis. CF is caused […] The post Eluforsen Improves CFTR Protein Function in CF Patients with F508del Mutations, Phase 1 Trial Shows appeared first on Cystic Fibrosis News Today. Read More WHO Approves Ensifentrine as Name for Potential Inhaled Treatment Now in Clinical Trials Jan 11, 2019 07:30 am | Jose Marques Lopes, PhD The World Health Organization (WHO) approved ensifentrine as the recommended name to identify the cystic fibrosis (CF) treatment candidate RPL554. Verona Pharma’s ensifentrine is an inhaled inhibitor of the enzymes phosphodiesterase 3 and 4, designed to have bronchodilator and anti-inflammatory properties. The “-fentrine” staple indicates WHO’s recognition that the compound inhibits multiple phosphodiesterases, the company reports. “We […] The post WHO Approves Ensifentrine as Name for Potential Inhaled Treatment Now in Clinical Trials appeared first on Cystic Fibrosis News Today. Read More New Inhaled Carrier May Work for CF mRNA Therapy, Mouse Study Finds Jan 10, 2019 07:00 am | Ana Pena An inhaled form of messenger RNA (mRNA) with the potential to safely deliver therapeutics to the lungs of cystic fibrosis (CF) patients has been discovered by researchers at the Massachusetts Institute of Technology (MIT). Researchers developed a type of nanoparticle given as an inhaled mist (nebulizer) that allows the delivery of mRNAs to the lungs of […] The post New Inhaled Carrier May Work for CF mRNA Therapy, Mouse Study Finds appeared first on Cystic Fibrosis News Today. Read More   Recent News Alcresta’s Relizorb Given Medicare/Medicaid Billing Code for Easier Claims My Trip to Hawaii Symbolizes My Victory Over Death by CF Vertex and Arbor Biotechnologies Collaborating to Develop Cystic Fibrosis Gene Therapies Empowering Children by Educating Them About Their CF Claire’s Place Foundation Grants to Help CF Families Reaches $1 Million Milestone