Food allergy prevalence was lower in those with cystic fibrosis vs. the general pediatric population. The prevalence of food allergy significantly differed by sex and pancreatic enzyme replacement ...

Sionna Therapeutics recently secured an initial public offering (IPO) of $191m on the Nasdaq stock exchange, signalling ...

Vertex Pharmaceuticals (NASDAQ:VRTX) announced Friday that an expert panel of the EU drug regulator, the European Medicines ...

Dr. Francis Collins, a renowned geneticist and former longtime director of the National Institutes of Health, announced ...

Once-daily vanzacaftor-tezacaftor-deutivacaftor was safe and well-tolerated in children with cystic fibrosis, while ...

Researchers trial inhalable gene therapy for cystic fibrosis, offering a potential breakthrough in treating the genetic lung ...

The trial specifically focuses on adults with CF who genetically cannot benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulators. 1 CF is a hereditary, lifelong disease that ...

Boehringer Ingelheim and partners have commenced the Phase I/II LENTICLAIR 1 trial of gene therapy BI 3720931 targeting ...

Parents are expressing concerns over an effort at the State House to eliminate the New Hampshire Vaccine Association, with some asking lawmakers to consider the plight of children who face significant ...

Private foundations providing support, resources, and advocacy for individuals and families affected by rare diseases have ...