News

March 16, 2020March 16, 2020

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

cystic fibrosis, News

A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of cystic fibrosis (CF) — in stem cells from CF patients, ... Read more

March 13, 2020March 13, 2020

Bonnell Foundation, Run by Michigan Mom of 2 CF Girls, Aims to Raise $1M

cystic fibrosis, News

When Laura Bonnell, founder and president of the Bonnell Foundation, was 12 years old, she decided to become a reporter. That dream led to a long career at WWJ, ... Read more

March 12, 2020March 12, 2020

Vertex Says It Can Deliver CF Medications Worldwide Despite COVID-19 Outbreak

cystic fibrosis, News

Vertex Pharmaceuticals said the worldwide COVID-19 outbreak has not affected its supply chain or its 2020 business outlook. The company said it’s confident it can continue to supply all ... Read more

March 11, 2020March 19, 2020

CF Families Advised to Take Preventive Measures to Avoid Coronavirus

cystic fibrosis, News

With the outbreak of coronavirus (COVID-19) affecting thousands of people around the world, individuals with respiratory conditions such as cystic fibrosis (CF) need to take precautions, according to medical expert ... Read more

March 10, 2020March 10, 2020

Nanoparticles with Plant Sterols May Aid Gene Therapy for CF

News

Tiny particles, or nanoparticles, work better as transport and delivery vehicles for the RNA molecules central to some forms of gene therapy when plant-based relatives of cholesterol, called phytosterols, ... Read more

March 9, 2020March 9, 2020

Topline Phase 2 Trial Data on ELX-02 Expected in First Half of 2020, Eloxx Says

cystic fibrosis, News

Eloxx Pharmaceuticals anticipates reporting topline data from the Phase 2 clinical trial program for ELX-02 — its lead therapeutic candidate to treat cystic fibrosis caused by nonsense mutations — ... Read more

March 6, 2020March 6, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

cystic fibrosis, News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

March 4, 2020March 5, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

News

In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the ... Read more

March 4, 2020March 4, 2020

Ultrasound Patterns May Predict CF Liver Disease, Study Suggests

cystic fibrosis, News

Liver ultrasounds can help identify children with cystic fibrosis (CF) who are at higher risk of developing liver disease, a new study shows. The study, “Heterogeneous Liver on Research ... Read more

March 3, 2020March 3, 2020

Respiratory Infection Candidate AB569 Kills Resistant Bacteria, Study Shows

News

AB569 is safe and effective at killing bacteria resistant to traditional antibiotics, in particular Pseudomonas aeruginosa (P. aeruginosa), an important disease-exacerbating microbe in cystic fibrosis (CF), a study showed. The study, “AB569, ... Read more