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…
Rare Disease Diversity Coalition Awards $600K to Combat Disparities
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…
When I learned that the prognosis for idiopathic pulmonary fibrosis (IPF) patients is about five years after diagnosis, I started wondering how my disease would progress. I didn’t think my progression would follow the usual, defined stages. I believed I would have an acute exacerbation and go directly from diagnosis…
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…
Every October, numerous causes host an awareness month. For instance, you are likely aware that it’s Breast Cancer Awareness Month. Thanks to widespread campaigns, pink ribbons have been popping up everywhere in my city and on virtual platforms like Facebook. A lesser-known but significant awareness month also occurs in…
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…
“But if these years have taught me anything it is this: you can never run away. Not ever. The only way out is in.” — Junot Díaz I have always been a social person. I need human contact and I have always enjoyed spending time with family and friends,…
