Cystic Fibrosis News Today Weekly Digest

Cystic Fibrosis Weekly Update Contents: Rare Disease Film Festival Highlights Patient and Researcher Unity Inspiration for Hire: Sick Candidates Only Proteostasis Therapeutics Completes Enrollment in Phase 2 Study Testing Triple Combination Therapy in Cystic Fibrosis Rare Disease Film Festival Highlights Patient and Researcher Unity Nov 13, 2019 07:00 am | Brad Dell Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare diseases that have been discovered; on […] The post Rare Disease Film Festival Highlights Patient and Researcher Unity appeared first on Cystic Fibrosis News Today. Read More Inspiration for Hire: Sick Candidates Only Nov 12, 2019 03:00 pm | Bailey Vincent At this time last week, I was crying. I was crying because I didn’t “want to let them down.” The potentially disappointed party in question was a new company within my professional dance company, aptly titled “Comebacks.” Initially, I wanted Comebacks to be the ultimate “comeback” for former dancers and athletes stricken by progressive illness. […] The post Inspiration for Hire: Sick Candidates Only appeared first on Cystic Fibrosis News Today. Read More Proteostasis Therapeutics Completes Enrollment in Phase 2 Study Testing Triple Combination Therapy in Cystic Fibrosis Nov 12, 2019 07:00 am | Iqra Mumal MSc Proteostasis Therapeutics (PTI) has completed patient enrollment in a Phase 2 clinical trial evaluating its cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies in cystic fibrosis (CF) patients who have at least one copy of the F508del mutation in the CFTR gene (the defective gene in CF patients). The most common mutation in CF patients is […] The post Proteostasis Therapeutics Completes Enrollment in Phase 2 Study Testing Triple Combination Therapy in Cystic Fibrosis appeared first on Cystic Fibrosis News Today. Read More Recent News Latinos on 2 Caribbean Islands Carry CFTR Mutations Seen in 1% of CF Patients Elsewhere, Study Says Addressing the Issues that Trikafta Benefits Will Bring Bionic Pancreas May Be Useful in Managing Blood Sugar Levels in Adults with CFRD, Small Study Says Eloxx Presents Positive Preclinical and Clinical Data of CF Therapy Candidate ELX-02 Preclinical Data Supports Effectiveness of ARO-ENaC, Arrowhead’s Investigational Therapy for CF