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…
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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…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
The inhalation of tiny plastic particles called polystyrene microplastics — an emerging pollutant that affects both terrestrial and aquatic ecosystems — was associated with the development of pulmonary fibrosis (PF) in healthy mice, a study shows. Notably, these microplastics were found to promote oxidative stress and damage to lung cells,…
Boehringer Ingelheim Canada has finished negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) — a partnership between the country’s provincial, territorial and federal governments — for Ofev (nintedanib), the company’s approved oral medication for progressive fibrosing interstitial lung diseases (PF-ILD). The pCPA conducts negotiations with drug manufacturers for treatments…
C21 (VP01), Vicore Pharma’s investigational oral therapy, safely and effectively improves or stabilizes lung function in previously untreated adults with idiopathic pulmonary fibrosis (IPF), according to interim data from the open-label Phase 2 AIR trial. “Based on the mechanism of action of C21, which has both vascular and…
An analysis of gene activity profiles in lung tissue has identified important drivers of idiopathic pulmonary fibrosis (IPF). The genes discovered in this integrative analysis may predict disease outcomes and be used as potential therapeutic targets for new anti-fibrotic treatments, according to researchers. The study, “Integrative…
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
