August - 2018

MECFA Blog

New Lung Cell Type Linked to Cystic Fibrosis Emerges From Mouse, Human Cell Atlas Studies

NEW YORK (GenomeWeb) – Two new studies that used single-cell RNA sequencing to profile tracheal or bronchial tissue from mice or humans...

Cystic Fibrosis Further Explained by Rare Cell Type Discovery

Investigators from the Broad Institute of MIT and Harvard and Massachusetts General Hospital (MGH) have discovered a rare cell type in...

New Gene Therapy Could Treat Cystic Fibrosis With One Dose

A new partnership in the UK will develop a gene therapy for cystic fibrosis that could treat the disease with a single dose. Cystic...

Acebilustat Seen to Prevent Pulmonary Exacerbations in CF Patients in Phase 2b Trial

Topline data from a Phase 2b trial showed that the investigative therapy acebilustat taken once daily can reduce the number and frequency...

Specific Gene Mutation Causes Severe CF in Georgian Jewish Patients, Study Reports

The Q359K/T360K gene mutation, which is commonly found in Jewish patients of Georgian descent, is associated with a particularly severe...

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...

Gene Mutations Linked to Other Lung Diseases May Contribute to Bronchiectasis, Study Suggests

Mutations in genes linked to other lung diseases, specifically cystic fibrosis and primary ciliary dyskinesia, may also contribute to...

Vertex Anticipates Completing Enrollment of Phase 3 Trials Testing Triple Combo Therapies

Vertex Pharmaceuticals plans to conclude the enrollment of patients in its Phase 3 programs involving its triple combo therapies to treat...

CF Babies More Likely to be Born Preterm, Weigh Less, Study Finds

Babies with cystic fibrosis (CF) are more likely to be born preterm — less than 37 weeks of pregnancy — and have significantly lower...

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