Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…
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PBI-4050, Prometic Life Sciences’ treatment candidate, can prevent lung fibrosis progression in patients with idiopathic pulmonary fibrosis (IPF) when used alone or in combination with Ofev (nintedanib), Phase 2/3 trial data shows. Data from the trial were the subject of an oral presentation at the American…
Investigational Pamrevlumab halts the progression of lung fibrosis and improves lung function in patients with idiopathic pulmonary fibrosis (IPF), new results from the Phase 2 PRAISE trial show. These results were recently presented in a series of poster presentations at the American Thoracic Society (ATS) 2018 in San Diego. “We…
Treatment with Ofev (nintedanib) may reduce the risk of death in patients with idiopathic pulmonary fibrosis (IPF), Boehringer Ingelheim’s clinical trials indicate. Boehringer presented its new results on the effectiveness, safety and tolerability of Ofev at ATS 2018, the American Thoracic Society’s annual conference, held in…
Two years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation (GDPR) — a tough new law that aims to protect the EU’s 512 million citizens, including rare disease patients, from having their medical records misused, sold, or subject to extortion by hackers, third…
Treatment candidate GLPG1690 prevented lung function decline in a Phase 2 clinical trial of idiopathic pulmonary fibrosis (IPF) patients, Belgium-based Galapagos announced. The research, “Safety, tolerability, pharmacokinetics, and pharmacodynamics of GLPG1690, a novel autotaxin inhibitor, to treat idiopathic pulmonary fibrosis (FLORA): a phase 2a randomised…
At the 2nd Annual IPF Summit in San Francisco this summer, academic and biopharma experts in idiopathic pulmonary fibrosis (IPF) will again gather to discuss the latest developments in the field. Taking place Aug. 20-22, the conference aims to close the translational gap between preclinical data and clinical outcomes, a…
Veracyte, a leading genomic diagnostics company, launched an Early Access Program to make its Envisia Genomic Classifier test available for idiopathic pulmonary fibrosis (IPF) patients, the company announced. Envisia Genomic Classifier was designed to improve the diagnosis of IPF and help ensure an appropriate treatment without requiring surgery. The…
The Pulmonary Fibrosis Foundation (PFF) has added 15 treatment sites to its nationwide Care Center Network (CCN) — including one each in Wisconsin and Iowa, states which previously did not have centers with recognized expertise in diagnosing and treating pulmonary fibrosis patients. “As the leading resource for the pulmonary fibrosis…
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…
