Stephen's Weekly CF Column

This past Saturday, Fedex brought a small unassuming box to the porch of my home. It didn’t take me long to open it and find Vertex’s newest drug, Orkambi, wrapped in its white and lime green colored box. Inside lay foil wrapped pills neatly labelled Sunday through Monday to…

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 followed by a swirling of excitement through the CF community and…

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 peppered with periods when I have been physically active with sports…

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 to assert dominance in the lung, inflicting chronic exacerbations that need…

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 in fact thousands of years old, how then has it persisted…

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 first time that those with CF who have two copies of…

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 ‘Salty Girls’ photo Series Challenges Notions of Beauty and Cystic Fibrosis…

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…

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…

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…