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Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

Novel selective blockers of the NOX4 enzyme could ease oxidative stress and the subsequent development of fibrosis in lungs and other organs, according to scientists at the University of Arizona. The potential treatments have been licensed to a startup called Fibronox to speed up their development. NOX4, or NADPH…

Lung cancer patients with underlying idiopathic pulmonary fibrosis (IPF) treated with proton radiation therapy live longer than those treated with X-ray irradiation, a study shows. Additionally, fewer occurrences of treatment-related lung complications were reported in proton therapy-treated patients. The study, “Preliminary result of definitive radiotherapy in patients with non-small cell…

Algernon Pharmaceuticals announced its plans to conduct a new in vivo animal study comparing its lead anti-fibrotic NP-251 compound with clinically relevant doses of both Esbriet (pirfenidone) and Ofev (nintedanib), two approved medicines for the treatment of idiopathic pulmonary fibrosis (IPF). IPF is a chronic lung disease characterized by a progressive and irreversible decline in…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

Poor sleep quality — assessed by full-night sleep evaluation and patient questionnaires — is linked to an overall reduced quality of life in individuals with idiopathic pulmonary fibrosis (IPF), according to a study. The study, “Quality of life in idiopathic pulmonary fibrosis: The impact of sleep disordered breathing,”…

Oral administration of omipalisib, an investigational inhibitor of PI3K and mTOR cellular signals, can reduce the activity of pro-fibrotic cells in the lungs of patients with idiopathic pulmonary fibrosis (IPF), results from a Phase 1 trial show. These findings support studies to further explore PI3K and mTOR signals as potential…