University of California7d
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
Rolling Out2d
Cystic fibrosis care made easier for parents
Learn how to navigate cystic fibrosis care with confidence, from treatments and school plans to building a supportive medical ...
ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis
ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA ...
McGill University Health Centre13d
Major funding for research project on cystic fibrosis
A new research project focusing on rare genetic mutations that are linked to cystic fibrosis just received $600,000 in ...
sciencex9d
Cystic fibrosis gene therapy programme gets green light with public funding
A groundbreaking gene therapy trial for cystic fibrosis (CF) will begin in March, thanks to a new grant from a government funding body. One hundred and thirty adults and children with CF will take ...
Morningstar3d
ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis
investigational mRNA therapy being developed to treat cystic fibrosis (CF). CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Although recent ...
pharmaphorum2d
Trial of Boehringer's cystic fibrosis gene therapy imminent
An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, hoping to become a first-in-class treatment for the genetic disorder.
Cystic fibrosis patients could get new 'once-a-day' pill this year
Cystic fibrosis (CF) patients could be given access to a new drug on the NHS by the end of the year if it is given the green ...
KATC9d
Cystic Fibrosis Foundation to recognize Acadiana’s top young professionals
The Cystic Fibrosis Foundation will host Acadiana’s Finest, a campaign to raise funds and awareness for cystic fibrosis and recognize the area’s top young professional leaders.