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…
Columns
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…
My past few articles have touched upon the application of stem cells for the treatment of cystic fibrosis (CF), and while some of these treatments demonstrate great promise for the future of CF, it is important to establish more necessary discourse. With my past articles discussing Embryonic Stem Cells (ESCs),…
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…
My last two articles have highlighted how Embryonic Stem Cells (ESCs), Induced Pluripotent Stem Cells (iPSCs), and Gestational Stem Cells have shaped regenerative medicine research for patients with cystic fibrosis (CF). In order to further delve into the function of stem cells, various approaches must be taken to…
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…
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