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The New England Journal of Medicine recently published findings regarding two, multinational, phase 3 studies on the administration of lumacaftor (VX-809) in combination with ivacaftor (VX-770; kayldeco). The study, titled, “Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR,” demonstrated the safety and…

There are several promising approaches designed to improve the life expectancy and quality of life of those with cystic fibrosis. With current methods aimed to aid CF symptoms, new treatments are constructed to help us gain a greater perspective on CF treatment options. Stem cells are poised to play a…

What We Hope For I have found myself chasing a deferred hope waiting for my health to change, while watching it steadily decline. The release of Kayldeco by Vertex Pharmaceuticals Inc. in 2012 was a demonstration that the next generation of medications in cystic fibrosis therapy could begin to…

Since I’ve started writing for Cystic Fibrosis News Today, I’ve received many messages and e-mails regarding the state of stem cell research and its impact on cystic fibrosis. Unfortunately, responding to these questions usually opened doors to more questions that could lead to some confusion. In order to confront the…

Organ donors are critical to the survival of patients whose organs experience severe damage through trauma and disease. However, on average twenty-one people die per day while waiting for a transplant. As a disease that features lung transplantation as a possible course of treatment, those in the cystic fibrosis patient…

In diseases like cystic fibrosis, the lungs undergo constant healing and remodeling due to chronic infections. To better understand the repair mechanisms the lungs go through in diseases like CF and COPD, scientists have paid great attention to studying cellular regeneration. Scientists at the University of Pennsylvania and Duke University have discovered…

Being informed on the science, drug developments, and public policy of CF is an excellent way to engage with the CF community and connect with your healthcare team. If you become easily overwhelmed by the churning waves of internet news outlets, I would like to extend you a small life-saver…

Despite major progress made in medicine, current treatments are increasingly unable to meet patient needs in a wide range of diseases. As our population ages, medicine is confronted with a great challenge of treating more complex diseases and injuries. Most current medical practices only manage patients’ symptoms with medications or devices.

The FDA approval of the CFTR modulator, Kayldeco, in 2012 changed the paradigm of CF healthcare by providing a therapy to address the underlying cause of the disease. The previous paradigm in CF care was limited to only treat the symptoms of CF, such as inflammation, bacterial infection and…

Reid D’Amico is a biomedical engineering PhD student with Cystic Fibrosis. He is currently pursuing PhD research to combine both engineering and pulmonary research, an emerging branch of regenerative medicine that he believes could play a major role in next-generation CF therapy. Reid plans on contributing to Cystic Fibrosis News Today in a…