Women With CF Use Combined Hormonal Contraceptives More Than Other Methods, Study Reports
Among women of childbearing age with cystic fibrosis (CF), the use of combined hormonal contraceptives containing both estrogen and...
Planned CHOICES Trial in Rare CF Mutations Given Regulatory Support
Proteostasis Therapeutics reported receiving scientific advice for its CHOICES clinical trial program, which is to test its personalized...
We Must Pay Closer Attention to the Global CF Population
Based on the stringent qualifications the Cystic Fibrosis Foundation uses to determine who has cystic fibrosis, approximately 70,000...
FDA puts Translate Bio’s rare disease mRNA drug on hold
The FDA has placed a clinical hold on the IND for Translate Bio’s mRNA treatment for ornithine transcarbamylase (OTC) deficiency, a...
The Bittersweet Downside of CFTR Modulators
I’ve been on Orkambi (lumacaftor/ivacaftor), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, for just over three...
#NACFC2018 – RPL554 Shows Promising Results in CF Studies
Positive data from preclinical and Phase 2a studies evaluating the investigational cystic fibrosis (CF) treatment RPL554 were presented...