News

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

Vertex Pharmaceuticals has signed a letter of intent with the pan-Canadian Pharmaceutical Alliance (pCPA), which is an agreement in principle to extend public reimbursement to Trikafta, a triple-combination therapy for eligible cystic fibrosis (CF) patients. The extension adds to the letter of intent (LOI) with the pCPA for…

AirPhysio, a handheld device designed to help clear the mucus that builds up in the lungs of people with cystic fibrosis (CF), is now available online in the U.S. through Life Wellness Healthcare. The Australian-based company said in a press release that the AirPhysio — which…

A two-day virtual event called BreatheCon will offer adults with cystic fibrosis (CF) an opportunity to connect and share their experiences through open and honest dialogue. Registration for the Sept. 24–25 event, which is hosted by the Cystic Fibrosis Foundation, is free online. “CF can be extremely…

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

A nonprofit’s entry in the Philadelphia Marathon, Team EE is looking for enthusiastic people to join it for the November race that will help to raise funds for the 10% of people whose cystic fibrosis (CF) is caused by nonsense mutations. As a member of the Emily’s Entourage marathon…

The COVID‐19 pandemic increased socioeconomic instability among people with cystic fibrosis (CF), mostly in terms of employment and ability to afford food, according to data from a single-center U.S. study. Notably, patients who were employed prior to the pandemic appeared to be the most affected, reporting job loss and concerns…

Increasing the levels of the SFPQ protein helped to restore expression and function of the CFTR protein in cells with a F508del mutation in the CFTR gene, the most common cause of cystic fibrosis (CF). Work also showed that SFPQ levels were unusually low in these cells, suggesting this protein “may play…

A new study is seeking to understand whether and how the symptoms of cystic fibrosis (CF) are affected by the menstrual cycle. To that end, CF patients ages 18 to 45, who have regular monthly periods, are being sought to participate in the aptly named MENSTRUAL study —…

The U.S. Food and Drug Administration (FDA) has given fast track designation to ELX-02, an investigational treatment for people with cystic fibrosis (CF) caused by nonsense mutations. This FDA designation aims to speed the development and regulatory review of potential treatments for serious or life-threatening diseases with an unmet medical…