Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases
Liminal BioSciences is stopping the clinical development of its investigative oral therapy fezagepras (PBI-4050) for idiopathic pulmonary fibrosis (IPF), based on interim pharmacological data from a Phase 1 clinical trial testing it at ascending doses in healthy volunteers. The decision comes less than a month after the company announced plans…
The first participant has been enrolled in a Phase 3 trial to evaluate the impact of United Therapeutics’ Tyvaso (inhaled treprostinil) on lung function in adults with idiopathic pulmonary fibrosis (IPF). The trial, TETON (NCT04708782), aims to recruit approximately 396 people ages 40 and older, who…
Managing symptoms is an ongoing challenge for chronically ill patients. Whether they’re caused by an exacerbation, disease progression, or environmental factors, it’s often difficult for patients to get quick relief, including from respiratory symptoms. Despite their name, rescue inhalers aren’t always effective in managing or improving pulmonary fibrosis…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…
Note: This story was updated June 11, 2021, to clarify the Chan Zuckerberg Initiative supported the Human Cell Atlas Project, in addition to other scientific initiatives. Researchers have created a comprehensive blueprint of healthy lung vascular cells, whose dysfunction is associated with pulmonary fibrosis (PF) and other lung…
When I write about my lung transplant, I honor my donor, for whom I am forever grateful. I didn’t know him, but I know he was a caring person. Last August, I was the lucky recipient of his lungs, which changed my life like nothing else has, or ever…
One of the most important blessings in life is having a strong support group. A support team may consist of family members, friends, or a community of people. My support team included three of the greatest people in my life: my husband, daughter, and mother. It is important…
Scientists have found a small molecule, called ISRIB, that was able to reduce fibrosis (scarring) and other disease-related features in mice with induced pulmonary fibrosis (PF). ISRIB successfully enhanced the repair process in lung cells that is altered in PF, especially…
Progressive lung diseases like pulmonary fibrosis cause ripple effects throughout the body. In PF, lung scarring prevents the organs from exchanging gases correctly, resulting in oxygen deficits known as hypoxemia and hypoxia. Chronic hypoxemia, or low oxygen levels in the blood, affects all parts of the body.
