Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…
A Phase 1 trial assessing how cudetaxestat, an investigational treatment for idiopathic pulmonary fibrosis (IPF), interacts with two approved IPF treatments, Ofev (nintedanib) and Esbriet (pirfenidone), is now enrolling healthy volunteers, Blade Therapeutics, the therapy’s developer, announced. The trial (NCT04939467) is aiming to enroll 86 healthy adults, ages 18 to 55.
An open inspection issue at a third-party testing facility has put on hold United Therapeutics’ application seeking the approval of Tyvaso DPI, an investigational dry powder inhaled formulation of treprostinil for pulmonary hypertension associated with interstitial lung disease (PH-ILD), a group of disorders…
Research examining the role that protein-RNA interactions play in promoting pulmonary fibrosis (PF) and other related conditions is being accelerated by a $1.8 million grant from the National Institutes of Health (NIH). The funding, granted to Robert Silvers, PhD, a Florida State University (FSU) assistant professor of chemistry and…
In a society that shuns openly speaking about death and dying, it’s hard to find words to grapple with the terror of the unknown and the threat of imminent grief. It can be even harder to find a space in which to utter them. When my mom, Holly,…
A new diagnostic tool, the Envisia Genomic Classifier, can help make doctors more confident and accurate in diagnosing idiopathic pulmonary fibrosis (IPF), a new survey suggests. Using the Envisa tool, by Veracyte, also may help in increasing the proportion of patients referred to proper treatment, the survey found.
A patient’s relationship with their physician can factor heavily in how the patient views healthcare. A relationship that inspires comfort, confidence, and trust is one to cherish. My pulmonologist announced recently that he is retiring, and I’m feeling a mixed bag of emotions about the news. As a patient with…
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…
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…
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