The U.S. Food and Drug Administration (FDA) has granted treprostinil orphan drug designation for the treatment of idiopathic pulmonary fibrosis (IPF). Orphan drug designation is given to therapies with the potential to substantially improve treatment for rare diseases (conditions affecting fewer than 200,000 people in the U.S.). The designation…
Treprostinil Earns FDA Orphan Drug Designation for Treating IPF
I recently celebrated another birthday, my fourth since being diagnosed with idiopathic pulmonary fibrosis (IPF). Prior to adding “rare disease patient” to my list of personal identities, I never cared much about birthdays. Now, I embrace them and enjoy celebrating the year that has passed and looking forward to…
The global COVID-19 pandemic has had a negative local impact on healthcare for most people in the U.K. with pulmonary fibrosis (PF), who have seen their medical appointments canceled, their pulmonary rehabilitation programs halted, and their health status decline as a result of lockdowns and other safety measures,…
The nonprofit Three Lakes Foundation and the healthcare incubator MATTER announced the four winners of the 2020 Innovation Challenge, an initiative that promotes advancements in the way pulmonary fibrosis (PF) is diagnosed, treated, and managed. Winners of the challenge will receive a monetary award…
GLPG1205, an investigational therapy by Galapagos, was found to be safe and well tolerated, with an ability to lower lung function decline in adults with idiopathic pulmonary fibrosis (IPF), according to results from a Phase 2 clinical trial. Top-line data from the study, called PINTA (…
A Phase 3 clinical trial evaluating Bellerophon Therapeutics’ investigational therapy INOpulse — inhaled nitric oxide — in patients with pulmonary fibrosis (PF) at risk of developing pulmonary hypertension (PH) has enrolled its first patient. Top-line data from the trial, called REBUILD (NCT03267108), are expected by 2022. “We…
I have always been uplifted and inspired by stories of hope, resilience, and people overcoming challenges. I would argue that many others feel the same way, because as humans, we all crave connection with one another, and stories are powerful tools that can unite us. In our current global health…
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…
