Cystic Fibrosis News Today Weekly Digest

Cystic Fibrosis Weekly Update Contents: Symkevi Approved in EU for CF Patients with Two F508del Mutations, or One Plus a Minimal Function Mutation Exciting Revelations from the NACFC 2018 Poster Sessions, Part 1 Vertex Announces Plans for Symdeko, Hopes for Triple Combos Now in Pivotal Trials Symkevi Approved in EU for CF Patients with Two F508del Mutations, or One Plus a Minimal Function Mutation Nov 02, 2018 09:00 am | Ana Pena Symkevi (tezacaftor/ivacaftor combo), by Vertex Pharmaceuticals, has been approved in the European Union for patients with cystic fibrosis (CF) ages 12 or older who carry F508del mutations. Importantly, the European Commission’s decision to grant marketing authorization makes Symkevi the first treatment in Europe for CF patients with only one F508del copy plus another mutation that confers minimal function to CFTR, the defective gene in […] The post Symkevi Approved in EU for CF Patients with Two F508del Mutations, or One Plus a Minimal Function Mutation appeared first on Cystic Fibrosis News Today. Read More Exciting Revelations from the NACFC 2018 Poster Sessions, Part 1 Nov 01, 2018 09:00 am | Tré LaRosa First in a series. The poster sessions at the North American Cystic Fibrosis Conference (NACFC) in Denver this month included many interesting branches of the wonderful world of CF research. In this column, I’ll share a short overview of an interesting poster from each topic to showcase promising developments. This is not intended to be […] The post Exciting Revelations from the NACFC 2018 Poster Sessions, Part 1 appeared first on Cystic Fibrosis News Today. Read More Vertex Announces Plans for Symdeko, Hopes for Triple Combos Now in Pivotal Trials Nov 01, 2018 07:30 am | Emma Yasinski Vertex Pharmaceuticals announced it plans to request by year’s end that the label for Symdeko (tezacaftor/ivacaftor and ivacaftor), its recently approved disease-modifying therapy for cystic fibrosis (CF), be expanded to include children as young as 6 years old, the company announced in a recent investor report. It also plans to request that Kalydeco (ivacaftor), its first treatment and one […] The post Vertex Announces Plans for Symdeko, Hopes for Triple Combos Now in Pivotal Trialsappeared first on Cystic Fibrosis News Today. Read More   Recent News Santhera Starting Phase 1b/2a Trial Testing Potential CF therapy POL6014 Why Are Our Doctors Not Discussing Herbal Supplements with Us? VX-445 Triple Combo Improves Lung Function in CF Patients with F508del Mutations, Phase 2 Trial Shows #NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach #NACFC2018 — Celtaxsys’ Acebilustat Delays Onset, Reduces Rate of Pulmonary Exacerbations in CF, Phase 2 Study Shows