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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...

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...

New CRISPR Tool Fixes CFTR Mutations in CF Patients’ Stem Cells, Study Finds

A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of cystic fibrosis...

Proteostasis On Track to Develop Organoids for Personalized Medicine Strategy for CF Patients with R

A project aimed at personalized medicine based on the use of organoids, or “mini-organs,” from cystic fibrosis (CF) patients with rare...

New Tool for Rapidly Analyzing CRISPR Edits Reveals Frequent Production of Unintended Edits

New method unmasks all DNA changes caused by CRISPR in two days vs two months WILMINGTON, DE — Amidst rising hopes for using CRISPR gene...

Gene Editing Tool Corrects CF-causing Mutations in Patient-derived Cells, Study Shows

Using the CRISPR gene editing tool, a team of researchers was able to permanently correct two mutations in the CFTR gene — mutations that...

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...

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...

How to Love Your Body, Even If It’s Genetically Abnormal

When we say “me” or “I,” what exactly are we referring to? Are we referring to our bodies, our minds, or the combination of the two? It’s...

Abeona Therapeutics to Report New Preclinical Data Demonstrating Therapeutic Potential of ABO-401 fo

NEW YORK and CLEVELAND, April 30, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and...

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