FDA Advisory Committee Gives Cystic Fibrosis Drug Kalydeco (ivacaftor) Thumbs-Up for Patients Six And Up With R117H Mutation

Vertex Pharmaceuticals Incorporated announced Tuesday that the U.S. Food and Drug Administration’s Pulmonary Allergy Drugs Advisory Committee (PADAC) voted 13-2 to recommend approval of Kalydeco (ivacaftor) in people with cystic fibrosis (CF) ages six and older who have the R117H mutation in the cystic fibrosis transmembrane regulatory (CFTR) gene,…

CF To Be Part of New Newborn Screening Requirements in PA

Governor Tom Corbett Every year, millions of babies in the U.S. undergo newborn screening to detect conditions that may gravely affect their health or survival in the long run. This effort is part of healthcare’s emphasis on early detection, accurate diagnoses, and prompt intervention in order to prevent juvenile…

NIH Awards $15.6 M to Support Parion Sciences’s CF Studies

The National Institutes of Health (NIH) recently awarded $15.6 million in grants to support research into mucolytic agents discovered by Parion Sciences, a company dedicated to creating novel therapies for pulmonary diseases, such as Cystic Fibrosis among others. The five-year grants were given to The University of North Carolina, Chapel Hill (UNC-CH) and…