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…
#ECRD2018 – Genome Editing Might Be ‘Cure’ for Rare Diseases But Ethical Guidelines Needed, Panel Says
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…
It’s really difficult for me to hear from others that they understand what I am going through. Despite the motive for this sentiment usually being supportive, I struggle to accept the words, because unless they also have IPF, others do not understand what I endure. I certainly…
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…
I shared in a recent column that I’m going through testing for lung transplant consideration. It has been challenging, but I am grateful because I know many pulmonary fibrosis patients do not have this opportunity. Being poked and prodded is exhausting, stressful, and sometimes painful. But it…
