News

Canadian patient advocates and leaders in the field of cystic fibrosis (CF) are meeting with Canadian government officials to lobby for changes to proposed regulatory reforms that are preventing CF patients from accessing Vertex’s new breakthrough therapy, Trikafta. Trikafta, Vertex’s most recent FDA-approved medication for the…

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, a study shows. The new approach has the ability to correct mutations without the need to excise the affected region,…

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, the CBS Radio affiliate in Detroit. One day while doing a United Way promotional piece at the University of Michigan…

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 its approved medications to patients with cystic fibrosis (CF) around the world. COVID-19 — an acronym for COrona…

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 Richard K. Mathis, MD. Mathis is a board member of Claire’s Place Foundation, a nonprofit organization that…

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, are included, scientists report. These findings are in the study, “Naturally-occurring cholesterol analogues in lipid nanoparticles…

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 — during the first half of 2020, the company has announced. “We are committed to ensuring full enrollment of our Phase…

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 lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

In 1960, a 19-year-old woman with cystic fibrosis (CF) made medical history when she gave birth after only 34 weeks of pregnancy. The patient died three months after delivery. In 1986, by which time the life expectancy of people with the disease had risen substantially, some 50 women with…

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 #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…