Stephen’s Weekly CF Column

July 24, 2015September 28, 2015

An Undeniable Hope Part 2: An Accumulation of Steps Led To Orkambi

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, ... Read more

July 10, 2015July 10, 2015

The Many Perspectives of the FDA Approval of ORKAMBI

Stephen's Weekly CF Column

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 ... Read more

July 6, 2015July 6, 2015

In CF, Can Playing a Sport or Biking Mean Less Time in the Hospital and More Time Enjoying Life?

Stephen's Weekly CF Column

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 ... Read more

June 29, 2015June 29, 2015

Cystic Fibrosis and the Bacteria Within

Stephen's Weekly CF Column

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 ... Read more

June 18, 2015June 18, 2015

Has Tuberculosis Contributed to the Global Rates of Cystic Fibrosis?

Stephen's Weekly CF Column

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 ... Read more

June 15, 2015August 31, 2015

A New Drug Being Developed by Vertex and Parion Seeks to Treat All CF Patients

Stephen's Weekly CF Column

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. ... Read more

June 5, 2015August 31, 2015

Orkambi, Genome Editing, and a Few ‘Salty Girls’

News, Stephen's Weekly CF Column

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 ... Read more

June 1, 2015August 31, 2015

The Safety and Efficacy of ORKAMBI (lumacaftor-ivacaftor) and the Next Steps In Improving the Drug

Stephen's Weekly CF Column

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, ... Read more

May 25, 2015August 31, 2015

Dum Spiro Spero: While I Breathe I Hope

News, Stephen's Weekly CF Column

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 ... Read more

May 12, 2015May 12, 2015

Salk Institute Stem Cell Discovery Could Eventually Help Repair Organs, Reduce Transplant Needs For CF Patients

News, Stephen's Weekly CF Column

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 ... Read more