People with a familial history of short telomere syndrome (STS) — a disorder involving shortened telomeres, or chromosome “caps” — may be at an increased risk of developing pulmonary fibrosis, even if they themselves do not have the mutation that causes STS, a new study highlights. “This has…
Long-term exposure to air pollutants — nitrogen dioxide, ozone, and fine particle matter — appears to greatly raise the risk of acute exacerbations, or sudden bouts of symptom worsening, in people with idiopathic pulmonary fibrosis (IPF), a study of patients in Greece reported. “Our findings show consistent positive associations” between…
Certain genetic variants appear to lead to better survival in people with idiopathic pulmonary fibrosis (IPF) who are on anti-fibrotics, suggesting that genetic testing may help to select patients for specific treatments, a real-world study found. Specifically, people carrying a variant of the DSP gene, known as the rs2076295…
A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
GED-0507, Nogra Pharma’s experimental therapy, significantly reduced the levels of pro-inflammatory and pro-scarring molecules, while suppressing the maturation of myofibroblasts — the main drivers of lung scarring (fibrosis) — to a similar or greater extent as two approved anti-fibrotic therapies. These findings, based on data from mice with induced…
Julie Halston, a Broadway theater veteran and comedian, has received the 2020 Isabelle Stevenson Tony Award for her longtime volunteer efforts for the Pulmonary Fibrosis Foundation (PFF). Halston, who is part of the PFF board, has raised more than $2 million for the organization. She also helped to bring…
Syndax Pharmaceuticals and Incyte are forming a global partnership to develop and commercialize axatilimab, an investigational therapy for idiopathic pulmonary fibrosis (IPF) and other diseases characterized by excessive scarring. The companies are planning to launch a proof-of-concept Phase 2 clinical trial to test axatilimab in IPF patients…
The levels of Sox9 — a molecule that promotes lung scarring, or fibrosis — are significantly higher in the lungs of both patients and mouse models of idiopathic pulmonary fibrosis (IPF) than in those of their healthy counterparts, a study shows. Excessive Sox9 levels were found to promote fibroblasts’ maturation…
Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…
A Phase 2 clinical trial evaluating Endeavor BioMedicines’ experimental oral therapy taladegib (ENV-101) in people with idiopathic pulmonary fibrosis (IPF), has dosed its first patient. The trial (NCT04968574) is enrolling adults, ages 40 and older, with mild-to-moderate IPF at one Australian site, with at least three more…
Get regular updates to your inbox.
