This year at the 28th Annual North American Cystic Fibrosis Conference (NACFC) in Atlanta, Vertex Pharmaceuticals Incorporated announced their ongoing efforts to treat the underlying cause of cystic fibrosis (CF) by focusing on mutations in the CFTR gene (short for cystic fibrosis transmembrane conductance receptor) through a combination of drugs.

Indiana-based University of Notre Dame, a Catholic research university, has just received a generous gift from a pair of prominent families to fund the institution’s Center for Rare and Neglected Diseases. This specialized center will now be known as the Boler-Parseghian Center for Rare and Neglected Diseases, in commemoration…

Pseudomonas aeruginosa Promising data of an anti-infective therapeutic to treat Cystic Fibrosis-associated lung infections was presented in the 28th Annual North American Cystic Fibrosis Conference, held last week in Atlanta, GA, by N8 Respiratory, LLC. showing the efficacy of its lead compound CSA-13 as an antimicrobial peptide…

While Cystic Fibrosis is considered to be a rare, “orphan” disease, affecting about 70,000 people in total worldwide, the support community that surrounds it is significantly larger, and works tirelessly to support fundraising and advocacy for the disease. Now, a new initiative by one of the world’s leading CF advocacy…

OrPro Therapeutics, Inc. is presenting the newest data concerning its leading pre-clinical therapeutic, ORP-100, at the 28th Annual North American Cystic Fibrosis Conference held October 9-11 in Atlanta. ORP-100 is being developed to treat cystic fibrosis by attacking the pathological, adhesive mucus found in patients’ lungs. According to a…

Case Western Reserve University faculty and clinicians are among those this weekend commemorating the discovery 25 years ago of the transmembrane conductance Cystic Fibrosis gene (CFTR) and reflecting on subsequent treatment advances that stemmed from the genetic breakthrough a quarter-century ago. The discovery was acheived in 1989 by a…

Belgium-based clinical-stage biotech, Galapagos, recently presented the findings of its novel and complementary corrector series that modulates the cystic fibrosis transmembrane conductance regulator (CFTR) at the North American Cystic Fibrosis Conference (NACFC) in Atlanta. During the two sessions led by Dr. Katja Conrath, the company disclosed preclinical data…

One of the approaches to developing novel treatments for difficult-to-manage conditions such as cystic fibrosis (CF) is taking FDA-approved, well-studied therapies and combining them with other drugs to produce more effective formulations. Along these lines, Vertex Pharmaceuticals has just announced several of their upcoming research and…

Cystic Fibrosis (CF), one of the most common genetic diseases among Caucasian children, is classified as an orphan disease, estimated to affect roughly 70,000 individuals worldwide, 30,000 of which live in the United States. While there are several subsidies that cater to research and development…

European leader in Data Driven Medicine, Sophia Genetics, has just launched a first-of-its-kind cystic fibrosis transmembrane conductance regulator (CFTR) in vitro diagnostic (IVD) solution, which will allow doctors to conduct complete CF studies in one next-generation sequencing trial. By providing a complete study of CFTR variants in a single NGS experiment, the company’s…