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…
For most of my adult life I was blissfully unaware of rare diseases. This lasted until 2016, when I was diagnosed with idiopathic pulmonary fibrosis (IPF), a life-threatening and progressive lung disease that affects about 200,000 Americans, with 50,000 new cases diagnosed annually in the U.S. Like many others,…
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…
The availability and quality of healthcare patients receive is often based on where they live and what they can afford. This is especially true for those among us with a rare disease. That’s what health equity is all about. It belongs under the umbrella term “social justice,” which encompasses a…
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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