News

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

The new documentary “Beyond Breathless,” spotlighting interstitial lung disease (ILD) patients and their families, will feature actress and producer Queen Latifah and former NY Yankee Bernie Williams. “Beyond Breathless” premieres on the…

The National Institutes of Health (NIH) has awarded a researcher at the University of Pennsylvania the K08 Award for his research in pulmonary fibrosis (PF), the Pulmonary Fibrosis Foundation (PFF) announced. PFF scholar Jeremy Katzen, MD, is the recipient of the NIH career development award for…

NeuroScientific Biopharmaceuticals, the Institute for Respiratory Health, and the University of Western Australia (UWA) have initiated a preclinical study assessing the potential of EmtinB and other related, lab-made compounds to treat post-COVID pulmonary fibrosis. Post-COVID fibrosis is irreversible scarring of the lungs…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Oral LYT-100 (deupirfenidone) was found to be safe and well-tolerated at all doses in an ongoing Phase 1 trial evaluating its potential to treat disorders involving inflammation and fibrosis, such as idiopathic pulmonary fibrosis (IPF), and disorders of lymphatic flow. “Based on these results, we plan to move…

The Three Lakes Foundation, a nonprofit supporting better ways of diagnosing and treating pulmonary fibrosis (PF), announced the release of “Second Wind,” a short documentary film highlighting the need for organ donation. The documentary tells the story of Nick Brown of Indiana and his years of struggle until…

The first patient has enrolled in a clinical trial to understand the effect of Ofev (nindetanib) on adults who developed pulmonary fibrosis (PF) following acute lung injury from COVID-19 infection. “A significant percentage of COVID-19 patients with acute lung injury may develop lung fibrosis based on clinical…

The Pulmonary Fibrosis Foundation (PFF), which supports research that advance understanding and better ways of treating pulmonary fibrosis (PF), is calling for submissions for next year’s PFF Scholars program for emerging scientists. Program winners will receive up to $50,000 over two years for PF research that may ultimately lead…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…