December 12, 2019December 10, 2019

I’m Sharing My Friend’s Story with Hope of a Triple-organ Transplant

Columns, Las Cosas de la Vida - a Column by Luisa Palazola

In this column, I want to share the story of a friend who needs a triple-organ transplant: lungs, liver, and kidneys. Before I delve into his experience, I’d like ... Read more

December 12, 2019December 12, 2019

CFF Offers Emotional Send-off to Retiring CEO Preston Campbell

cystic fibrosis, News

Preston W. Campbell III, MD, recently bid farewell as president and CEO of the Cystic Fibrosis Foundation (CFF) in Nashville — the same city where he began his long ... Read more

December 11, 2019December 11, 2019

European Commission Approves Kalydeco for Children as Young as Six Months

cystic fibrosis, News

The European Commission has approved a label extension for Kalydeco (ivacaftor), allowing the therapy to be used in infants with cystic fibrosis (CF) who are between the ages of ... Read more

December 10, 2019December 6, 2019

Confessions from the 10 Percent: Life Without Trikafta

Columns, Life in the Grey: a Column by Bailey Anne Vincent

I am one of the 10 percent. The 10 percent cannot take the new, groundbreaking medication that will hopefully help cystic fibrosis (CF) patients for years to come. Some ... Read more

December 10, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

cystic fibrosis

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 10, 2019December 10, 2019

Kalydeco Improves Lung Function in Patient Carrying Responsive and Non-responsive Mutations, Case Study Reports

cystic fibrosis, News

Treatment with Kalydeco (ivacaftor) alone improved lung function and quality of life in a cystic fibrosis patient carrying both a therapy-responsive and a non-responsive mutation to treatment, a case study ... Read more

December 9, 2019December 5, 2019

We Must Build a Diverse Coalition Within the CF Community

Columns, Mutations & Conversations - a Column by Tré LaRosa

I learned long ago not to expect everyone to agree with me. I also realized that disagreement isn’t inherently wrong. From a young age, I was told that I ... Read more

December 9, 2019December 9, 2019

Copeptin a Potential Biomarker of Symptom Severity During Pulmonary Exacerbation, Study Shows

cystic fibrosis

High levels of the peptide copeptin are associated with increased severity of cystic fibrosis (CF) in children, a study shows. These results suggest that copeptin can be used as ... Read more

December 6, 2019December 6, 2019

Quebec Loans Laurent $2.7M to Finance Trial Testing Oral LAU-7b in All CF Types

cystic fibrosis, News

The Government of Quebec, in Canada, has approved a loan of $2.7 million Canadian (about $2.05 million U.S.) to Laurent Pharmaceuticals to help the company conduct a Phase 2 clinical ... Read more

December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 5, 2019December 5, 2019

Israeli, US Researchers Develop New Device to Eliminate Mucus Secretion from Obstructed Airways

cystic fibrosis, News

Researchers in Israel and the U.S. have developed a new technology that can be used to unclog and eliminate mucus secretions from the airways of patients with various respiratory ... Read more

December 4, 2019December 4, 2019

Cystic Fibrosis Canada Opens Holiday ‘Wish’ Fundraising Campaign

cystic fibrosis, News

To heighten cystic fibrosis (CF) awareness and raise funds for research, care and advocacy, Cystic Fibrosis Canada (CFC) has launched its fourth annual year-end campaign. This year’s effort, which began ... Read more