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December 13, 2019December 13, 2019

Proteostasis to Test Personalized Treatment with Novel CFTR Modulators in Europe

cystic fibrosis, News

Three investigative therapies by Proteostasis Therapeutics — PTI-801, PTI-808, and PTI-428 — showed potential to treat cystic fibrosis (CF) patients ineligible for approved CFTR modulators after patient-specific lab models ... 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 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 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

December 3, 2019December 3, 2019

Elexacaftor Triple Combo Improves Lung Function and Quality of Life in CF Patients, Phase 3 Trial Shows

cystic fibrosis, News

The therapeutic triple combo elexacaftor (VX-445), tezacaftor, and ivacaftor leads to marked improvements in lung function and quality of life in people with cystic fibrosis (CF) carrying the F508del mutation, data ... Read more

December 2, 2019December 2, 2019

CFF Strives to Improve Lung Transplant Journey for Cystic Fibrosis Patients

cystic fibrosis, News

When it comes to lung transplants, the Cystic Fibrosis Foundation (CFF) admits that for a long time the issue was shrouded in confusion, frustration, and lack of information. But ... Read more

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