Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…
Pliant Therapeutics will start testing, in trial, a higher dose of its investigational oral small molecule PLN-74809, an anti-fibrotic therapy for idiopathic pulmonary fibrosis (IPF). The higher dose of 320 mg will be given to about 28 adults with IPF once daily for at least six months as…
The investigational oral therapy Haduvio (nalbuphine extended-release tablets) significantly reduced chronic cough in people with idiopathic pulmonary fibrosis (IPF), according to interim results from the proof-of-concept Phase 2 CANAL trial. Findings in 26 patients showed a mean 51.7% placebo-adjusted decline in daytime cough frequency with the treatment, meeting the…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
The U.S. Food and Drug Administration (FDA) has given breakthrough status to Boehringer Ingelheim’s BI-1015550, an experimental oral therapy for idiopathic pulmonary fibrosis (IPF), based partly on the findings of a clinical trial in IPF patients. A breakthrough therapy designation works to speed the development and review of…
Joint infection with a virus and bacteria in people with idiopathic pulmonary fibrosis (IPF) significantly raises their risk of dying — by more than eight times — relative to patients with no such infections, a study reported. Viral plus bacterial infections were linked to acute exacerbations and disease progression in…
The approved antibiotic nifuroxazide halted the development, and reversed the signs, of induced pulmonary fibrosis (PF) in mice, a study showed. These data support further research into the repurposing of nifuroxazide as a viable therapeutic option for people with PF, according to scientists. The team noted that there were…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
People carrying a genetic variant of the gene coding for Mucin 5B — a component of lung mucus and needed for cellular function — are at increased risk of developing idiopathic pulmonary fibrosis (IPF), according to a recent study. In this variant, the nucleotide guanine, one of the building…
Two protein kinases, called DCLK1 and STK33, may serve as candidates for a molecular targeted therapy of idiopathic pulmonary fibrosis (IPF), a study reported. Current anti-fibrotic therapies for IPF have been shown to slow disease progression. One such approved treatment, Ofev (nintedanib), works by blocking the tyrosine kinase. But…
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