Patrícia Silva, PhD,  director of science content—

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…

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…

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…

The non-profit organization Cystic Fibrosis Research, Inc. (CFRI) is organizing a wine tasting event to help support research to find a cure for cystic fibrosis (CF), taking place on Sunday, October 19, between 3 and 7 pm at the Purple Orchid Wine Country Resort & Spa.

The British Gene Therapy Consortium announced recently that it has decided not to attend the upcoming North American Cystic Fibrosis Conference (NACFC), where they were planning on presenting the latest data about their Multidose Gene Therapy (Wave 1) Trial, which focuses on developing a genetic treatment for cystic fibrosis (CF).

Enterovirus D68, a non-polio enterovirus that causes mild to severe respiratory illness and can be spread through coughs, sneezes, or touch, has become a contagious problem in the United States. As a result, physicians are paying special attention to patients who already suffer from respiratory diseases or severe disease, such as cystic…

The Great Strides event, a fundraiser associated with the Cystic Fibrosis Foundation, has become the largest national fundraiser advocating for cystic fibrosis (CF), held through monthly initiatives and with the support of families, donors, and friends. This month, those honored by the event are Jen and Justin Grady,…