On Thursday, July 2, the Food and Drug Administration made the announcement that Orkambi had been approved for people with cystic fibrosis who are age twelve and older with two copies of the F508del mutation. The announcement was…
There are strains of mice in the lab that can run for what seems an endless amount of time. If I happened to be a mouse in a lab I would not be that mouse. My life has been…
Cystic fibrosis is a complex genetic disease that slowly takes the breath of those who have it by eroding the function of their lungs over time. At the core of this erosion is a diverse community of bacteria competing…
Cystic Fibrosis is not a disease new to modern history. It is speculated that people have died from the disease for hundreds if not thousands of years. If cystic fibrosis, which is terminal in the majority of patients, is…
The CF community waits with warm hearts and open arms for the approval of ORKAMBI, the combination drug by Vertex Pharmaceuticals, slated to be approved next month on July 5. The excitement is well deserved because it is the…
The selection of articles I chose for this week is an attempt to capture recent events, along with one older article, showcasing the range of articles relating to the science and increased awareness being brought to the CF community…
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…
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…
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…
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…
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…
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