miércoles, 25 de septiembre de 2019

Cystic Fibrosis News Today Weekly Digest

Cystic Fibrosis Weekly Update

Contents:

CF Foundation Awards Eloxx $1.61M for Phase 2 Trials in People with Nonsense Mutations

Sep 25, 2019 07:00 am | Joana Carvalho, MSc



CFF awardThe Cystic Fibrosis Foundation (CFF) is giving Eloxx Pharmaceuticals up to $1.61 million to support its planned Phase 2 clinical trial program assessing the safety, tolerability, and chemical properties of ELX-02, Eloxx’s lead investigational compound to treat cystic fibrosis (CF) caused by nonsense, or stop, mutations. The program is set to include two open-label, dose escalation, Phase […]
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I Want to Break Up with My CF Clinic

Sep 24, 2019 03:00 pm | Bailey Vincent



CF clinicI might break up with my CF clinic. It’s not them, it’s me. They are doing a good job. My CF doctor is fine. My nurses, dietitian, and support staff are really cool. And everything is basically OK. They’re nice people. It’s fine. The problem is, I’m not sure what the clinic is really doing […]
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Kalydeco’s Efficacy Tied to Phosphorylation Levels of CFTR Protein, Study Suggests

Sep 24, 2019 07:00 am | Patricia Inacio, PhD



Kalydeco studyCFTR channel activity is controlled by a molecular process known as phosphorylation. Now, a study suggests that the levels of phosphorylation affect how well Kalydeco works in people with certain mutations in the CFTR gene. The study “VX-770-mediated potentiation of numerous human CFTR disease mutants is influenced by phosphorylation level” was published in the journal Nature Scientific Reports. […]
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