The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…
RXC007, an investigational oral therapy for idiopathic pulmonary fibrosis (IPF), is showing favorable safety and pharmacological properties in an ongoing Phase 1 trial in healthy volunteers. The experimental therapy being developed by Redx Pharma works by blocking the activity of ROCK2, a protein involved in a signaling cascade that…
People with idiopathic pulmonary fibrosis (IPF) have an eight times higher risk of mortality during hospitalizations or within three months of being discharged than outside this time window, a U.S. study has found. The risks were even greater for patients who were admitted to the hospital for a respiratory…
Treatment with N115, EmphyCorp’s non-steroidal nasal spray, led to statistically and clinically significant improvements across all lung function parameters in patients with pulmonary fibrosis (PF) participating in a Phase 3 trial, data show. Now, data from two other Phase 3 clinical trials suggest that N115 also may be…
Note: This story was updated Oct. 22, 2021, to correct information about the study that was wrongly identified as a different clinical trial. A new study is hoping to identify early drivers of pulmonary fibrosis (PF) in individuals with a family history of PF, with the goal of…
A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.
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…
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