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As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…

A new lung-on-a-chip technology models the mechanical changes that occur in the lungs during idiopathic pulmonary fibrosis, possibly allowing for a faster and less expensive screening of potential new anti-fibrotic therapies, a study reports. “Obviously it’s not an entire lung, but the technology can mimic the damaging effects of lung…

A pulmonologist’s characteristics — such as the nature of the practice and previous treatment experience — can influence when anti-fibrotic therapy use is initiated in newly diagnosed idiopathic pulmonary fibrosis (IPF)  patients, a study based on a U.S. survey found. This finding adds to an understanding of the factors, besides patient preference,…

Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of genome editing to somatic cells, so that they don’t pass changes on to the next generation? What about stem-cell research, three-parent…

Seasonal flares of disease activity that can lead to idiopathic pulmonary fibrosis (IPF) patients being hospitalized, especially in the winter months, appear to be effectively countered by treatment with Esbriet (pirfenidone) compared to placebo, a new retrospective analysis of data from Phase 3 clinical trials shows. Results were presented by Brett Ley,…

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…

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…