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MECFA Blog

CHMP Gives Positive Opinion to Vertex’s Triple Combination Therapy Kaftrio

The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in...

Brensocatib prolongs time to first pulmonary exacerbation in patients with bronchiectasis

Treatment with brensocatib for 24 weeks significantly prolonged time to first pulmonary exacerbation in patients with non-cystic fibrosis...

New Combination of CFTR Mutations Found in Chinese Siblings With CF

A combination of two mutations, including one that had never been reported, were found in two Chinese siblings who developed a severe...

Second-hand Smoke Affects Metabolism in Infants and Children with CF, Study Finds

Exposure to second-hand smoke in infants and children with cystic fibrosis (CF) is linked to changes in levels of metabolites and...

Cystic Fibrosis and Herpes Are Not the Same Thing

I don’t have herpes. I need to clarify this. I don’t have herpes, or Hep C, or cancer, even though many seem to think I do. Although I...

4DMT Receives Up to $14M from CFF for Development of Customized Gene Carrier

After being given more than $3 million from the Cystic Fibrosis Foundation (CFF) in 2017 to expand the development of viral vectors for...

Bacterial 'Death Stars' could be tricked into destroying themselves

Researchers have discovered a network of channels inside bacterial communities which could be used to kill bacteria more quickly by...

Suppressing Function of Chloride Channel TMEM16A Eyed as Potential Therapeutic Approach

Researchers have reported the case of two siblings with impaired CFTR function due to a deficiency in another chloride channel, called...

Microbe DNA Discovery May Lead to Noninvasive Test for CF Infections

People with cystic fibrosis (CF) have higher levels of DNA from common microbes that cause CF-related infections circulating in their...

Real-world Use of Kalydeco for CF Highly Effective, Large-scale Analysis Shows

Kalydeco treatment led to fewer deaths, transplants, hospitalizations, and exacerbations in people with cystic fibrosis (CF) compared to...

Disease Duration, Flare-ups Affect Mental Health of Non-CF Bronchiectasis Patients, Study Finds

VERTEX’S CF BLOCKBUSTER TRIKAFTA EFFECTIVE IN KIDS 6 TO 11 YEARS OF AGE

A case for improved carrier screening

Vertex to Seek Expanded FDA Approval for Trikafta After Positive Child Safety Data

Vertex’s Kaftrio approved for use in Europe and made accessible on the NHS

Age May Affect Outcomes in Lung Transplant Recipients With Cystic Fibrosis

CFF Funding Will Further ContraFect’s Research Into Alternative for Antibiotics

The search for molecular glue in targeted disease control

Vest Therapy Lowers Risk of Hospitalization in Non-CF Bronchiectasis, Study Says

Harvard is working with Boston hospitals to test existing cystic fibrosis drug to help coronavirus p