News

Novoclem Therapeutics, which is developing nitric oxide-releasing compounds that fight infections in cystic fibrosis and other respiratory diseases, has named five inaugural members of its scientific advisory board. They are Dr. Donald VanDevanter, adjunct professor at Case Western Reserve University School of Medicine; Dr. Felix Ratjen, a professor of pediatrics at the…

Relizorb, an enzyme-containing cartridge that breaks down fats in tube-feeding formulas, is now available to children, according to its developer, Alcresta Therapeutics. The U.S. Food and Drug Administration, which approved the device’s use in adults in 2015, has widened the approval to children as young as 5 years old, Alcresta said The…

In September 2016, Jerry Cahill and Emily Schaller joined a high-profile effort to persuade cystic fibrosis patients like themselves to cycle to improve their health. The title of the event said it all: It was the third annual Bike to Breathe adventure. Convinced that cycling helps them combat a disease…

The U.S. Food and Drug Administration (FDA) has approved Digestive Care’s Pertzye (pancrelipase) to be given to cystic fibrosis patients through a so-called G-tube — a feeding tube delivering nutrition directly to the stomach through a hole in the abdomen. Pertzye is an enzyme replacement therapy for exocrine pancreatic…

Three triple-combination treatments from Vertex Pharmaceuticals showed promising results in Phase 1 and Phase 2 clinical trials in cystic fibrosis (CF) patients with one F508del mutation and one so-called minimal function mutation (F508del/Min) in the CFTR gene, the defective gene that causes CF. Vertex announced that these are the first data to demonstrate…

Results of an online survey, conducted by the cystic fibrosis (CF) community GeneFo, showed that a main concern of CF patients is their intimate lives and sexuality. In an attempt to help CF patients and caregivers to find ways, alone or as a couple, to overcome the physical…

The Italian health insurance system has agreed to cover cystic fibrosis patients’ use of Vertex Pharmaceuticals‘ Orkambi (lumacaftor/ivacaftor). Those eligible for the Italian Medicines Agency coverage are patients 12 and older who have two copies of a particular mutation of the gene that is defective in the disease. That is the…

The U.S. Food and Drug Administration is promoting a targeted treatment approach known as precision medicine to help people with diseases stemming from specific, and often rare, genetic features. Its efforts include expanding the use of approved therapies to other genetic-based conditions, and pushing for the development of more biomarkers…

Synspira’s SNSP113 gives antibiotics more punch against the Burkholderia cepacia infections that cystic fibrosis patients experience, a study reports. The research, published in PLoS One, was titled “Novel glycopolymer sensitizes Burkholderia cepacia complex isolates from cystic fibrosis patients to tobramycin and meropenem.” Lung infections caused by Burkholderia cepacia are difficult to…

A first-grader with cystic fibrosis (CF), Conor Fink, inspired a close family friend to hop on his bicycle and spend his summer pedaling from the West to East Coast — Oregon to New York — to  raise money for Claire’s Place Foundation, a nonprofit helping children and families affected by the disease.