Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
Rare Disease Day at NIH, Set for March 1, Growing Year by Year
The University of South Florida (USF) received a $1 million donation to fund research focused on finding ways to detect and prevent lung tissue scarring in people with pulmonary fibrosis (PF). “This generous gift will help the University of South Florida advance innovative testing and treatment of the toughest…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Since the start of the pandemic, levels of stress, frustration, and confusion have risen to troubling levels. It seems that many of us also are experiencing more concern over new, rapidly spreading coronavirus variants. When I first heard about the variants, I broke down and cried. I felt defeated…
A new lung-on-a-chip recreates complex lung structures using a patient’s own cells, providing a more precise model for studying disorders such as pulmonary fibrosis (PF), a study reported. This second-generation model is expected to give researchers with a powerful new way to conduct basic lung research, and a faster,…
The American Lung Association (ALA) gave a Catalyst Award, worth $100,000 over two years, to a scientist whose study will address the regeneration of damaged lung tissue. Ya-Wen Chen, PhD, an assistant professor of medicine and stem cell biology and regenerative medicine at the University Southern California (USC), will…
Galapagos and Gilead Sciences have suspended the worldwide ISABELA Phase 3 program investigating the potential of oral ziritaxestat (GLPG1690) to treat people with idiopathic pulmonary fibrosis (IPF). The decision to halt the program, which includes two identical Phase 3 trials — ISABELA 1 (NCT03711162) and ISABELA 2…
Last month we moved into our new home in Hawaii. My husband, Jonny, flew back to the mainland immediately for several months of Army training, so I was left to get settled on my own. I started tearing through all the boxes and piles of things to make the place…
The U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for ImmunoMet Therapeutics‘ treatment candidate for idiopathic pulmonary fibrosis (IPF), IM156. The decision grants ImmunoMet permission to open studies of the therapy in people in the U.S. “FDA clearance of an IND for…
The U.S. Food and Drug Administration (FDA) has accepted and granted priority review to an application for Esbriet (pirfenidone) as a treatment for unclassifiable interstitial lung disease (UILD). The supplemental new drug application was submitted by Genentech, Esbriet’s developer and a member of the Roche Group, based on…
