June - 2019

MECFA Blog

FDA Approves Symdeko for Treatment of CF Children, Ages 6 to 11, with Certain CFTR Mutations

The U.S. Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) tablets for the treatment of...

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those...

Eluforsen Found to be Safe for Adults with Cystic Fibrosis Caused by F508del Mutation, Study Reports

The experimental treatment eluforsen is safe for adults with cystic fibrosis(CF) caused by a mutation called F508del, a new study...

Murepavadin Antibiotic Well-tolerated by Animals Infected with Pseudomonas Aeruginosa, Preclinical D

Murepavadin (POL7080), Polyphor‘s lead investigational antibiotic targeting Gram-negative bacteria, behaved as expected and was...

EffRx Pharmaceuticals Signs Exclusive License Agreement with Pharmaxis for the Registration and Comm

EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today...

Slammed by Side Effects: The Downside of Antibiotics

Everyone with cystic fibrosis knows that feeling. Sometimes, it creeps up on you slowly. Other times, it hits right away, like a truck...

New study confirms vaping is not safer than smoking- it obstructs mucus clearance

Research published in the American Thoracic Society’s American Journal of Respiratory and Critical Care Medicine confirms that...

Trial of Nebulized CF Candidate Therapy Completes Single-ascending Dosing

The RESTORE-CF clinical trial investigating Translate Bio’s candidate therapy MRT5005 has completed the single-ascending dose regimen in...

Specific Peptide May Lower Antibiotic Resistance of P. aeruginosa Bacteria, Study Reports

Expression of a peptide called RpoN* in antibiotic-resistant Pseudomonas aeruginosa isolates from cystic fibrosis (CF) patients made the...

Life-saving designer ‘NINJA virus’ hailed as medical breakthrough in curing infection

A VIRUS cocktail designed to seek and destroy bacteria has cleared a 15-year-old lung transplant patient of an antibiotic-resistant...

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