Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
Four years of monitoring clinical use of Ofev (nintedanib) by people with idiopathic pulmonary fibrosis (IPF) has found no new safety concerns, a database study found. Treatment safety and tolerability were consistent with findings reported in pivotal trials, with non-severe diarrhea continuing to be the most common side effect noted,…
People with idiopathic pulmonary fibrosis (IPF) who are treated with either Ofev (nintedanib) or Esbriet (pirfenidone) report similar satisfaction with their medications, a new study suggests. The study, “Patient expectations, experiences and satisfaction with nintedanib and pirfenidone in idiopathic pulmonary fibrosis: a quantitative study,”…
NXP004, Nuformix’s potential anti-fibrotic treatment for idiopathic pulmonary fibrosis (IPF), led to a dose-dependent reduction in the production and deposition of proteins that are known key drivers of fibrosis (tissue scarring) and IPF progression, a preclinical study shows. Although these are preliminary findings in a tissue study,…
Adelmidrol therapy reduced markers for inflammation and oxidative stress, as well as tissue damage and collagen deposits, in the lungs of mice with pulmonary fibrosis (PF), a study has found. The findings support further investigation of adelmidrol as a…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
The world must learn to address our widespread anxiety about the ongoing health crisis. As a patient living with idiopathic pulmonary fibrosis (IPF), a life-threatening lung disease that makes me increasingly vulnerable to respiratory infections, I’m familiar with this anxiety. But life must continue, and it’s necessary to find a…
A gene therapy that lengthens the life of lung cells can halt scarring and help sustain lung regeneration in a mouse model of age-related pulmonary fibrosis, a new study shows. The study, “Telomerase treatment prevents lung profibrotic pathologies associated with physiological aging,” was published in the Journal…
The nonprofit Three Lakes Foundation has teamed with Matter, a healthcare incubator, to launch the PF Innovation Challenge, an effort to promote better ways of diagnosing pulmonary fibrosis (PF) and caring for patients. Together, these Chicago-based groups set a total of $100,000 aside for the winning approaches. “Over the…
The first participant has been enrolled in a Phase 3 clinical trial evaluating Ofev (nintedanib) in children with fibrosing interstitial lung diseases (ILDs). The Phase 3 trial, called INPEDILD (NCT04093024), is sponsored by Boehringer Ingelheim, Ofev’s manufacturer. The trial is currently recruiting participants from 6–17 years…
