cystic fibrosis

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

When it comes to lung transplants, a study has found that the practice of many cystic fibrosis (CF) specialists differs from that of updated recommendations, highlighting a need to educate providers on new guidelines. The study, “Lung transplant referral practice patterns: a survey of cystic fibrosis physicians and general…

Savara Pharmaceuticals has stopped patient enrollment into two of its ongoing clinical trials in cystic fibrosis (CF) patients — AVAIL and ENCORE — for safety reasons due to the COVID-19 pandemic. Steps will also be taken to ensure that those already enrolled in both studies continue…

ReCode Therapeutics has raised $80 million in Series A financing to support the preclinical development of its lead targeted RNA therapy for cystic fibrosis (CF). The funding also will be used to support the development of RNA therapies for primary ciliary dyskinesia (PCD), a hereditary condition that impairs the function…

Translate Bio‘s therapy MRT5005 has been given a rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of cystic fibrosis in children, while its ongoing clinical trial investigating the therapy in adult patients faces delays due to the COVID-19…

Vertex Pharmaceuticals is “highly confident” that it can continue to supply its approved cystic fibrosis (CF) therapies to patients worldwide, but expects at least some of its ongoing and planned clinical trials to be affected by the COVID-19 pandemic. Among likely changes to ongoing trials well into the dosing of patients — including…

At 3 a.m. on July 6, 2003, Miami resident Jeffrey Goldstein got a call that would change his life. Doctors at the Duke University Lung Transplant Clinic had found a lung donor match for Goldstein, who had been diagnosed with pulmonary fibrosis two years earlier. Nearly 17 years…

Eloxx Pharmaceuticals has temporarily paused enrollment in Phase 2 clinical trials of ELX-02 in cystic fibrosis (CF) patients in response to the COVID-19 pandemic. According to Eloxx,…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD), said it will still take place at the…

Problems with the CFTR protein in platelets, blood cells essential to clotting, activated these cells, both driving lung inflammation and injury, and hindering the lungs’ ability to clear bacteria in a mouse models of cystic fibrosis (CF), a study found. Blocking a calcium channel known as TRPC6 returned platelets…