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MECFA Blog

Women With CF Use Combined Hormonal Contraceptives More Than Other Methods, Study Reports

Among women of childbearing age with cystic fibrosis (CF), the use of combined hormonal contraceptives containing both estrogen and...

Planned CHOICES Trial in Rare CF Mutations Given Regulatory Support

Proteostasis Therapeutics reported receiving scientific advice for its CHOICES clinical trial program, which is to test its personalized...

We Must Pay Closer Attention to the Global CF Population

Based on the stringent qualifications the Cystic Fibrosis Foundation uses to determine who has cystic fibrosis, approximately 70,000...

FDA puts Translate Bio’s rare disease mRNA drug on hold

The FDA has placed a clinical hold on the IND for Translate Bio’s mRNA treatment for ornithine transcarbamylase (OTC) deficiency, a...

The Bittersweet Downside of CFTR Modulators

I’ve been on Orkambi (lumacaftor/ivacaftor), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, for just over three...

#NACFC2018 – RPL554 Shows Promising Results in CF Studies

Positive data from preclinical and Phase 2a studies evaluating the investigational cystic fibrosis (CF) treatment RPL554 were presented...

Vertex and Treating CF: High Hopes for Triple Combinations and Gene Therapy Possibilities

Editor’s note: This is third in a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we look at...

Orkambi Not Seen to Significantly Improve Blood Glucose Levels in CF Patients in Small Study

Analysis of blood glucose levels in cystic fibrosis (CF) patients being treated with Orkambi (ivacaftor/lumacaftor) shows that the...

Cystic Fibrosis Treatment Approved for Use in Children Aged 2 to 5 Years

Officials with the FDA have updated the label of lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals), approving the treatment's use in...

Vertex Pharma (VRTX) Receives European CHMP Positive Opinion for SYMKEVI for People with CF Aged 12

Vertex Pharmaceuticals (Europe) Limited (Nasdaq: VRTX), today announced that the European Medicines Agency’s (EMA) Committee for...

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