News

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

The European Idiopathic Pulmonary Fibrosis (IPF) and Related Disorders Federation (EU-IPFF) is welcoming patients, clinicians, researchers, industry representatives, and policymakers to register for a three-day virtual summit  regarding pulmonary fibrosis (PF) and other interstitial lung diseases (ILDs). The European PF Patient Summit, which will be held online April…

People with idiopathic pulmonary fibrosis (IPF) in poorer health are less likely than healthier patients to tolerate more than one year of treatment with Ofev (nintedanib), and benefit less from the therapy than those able to continue with its use, a small real-world study reports. Side effects that…

In mouse models of lung cancer and fibrosis, a combination of Esbriet (pirfenidone) and anti-PD-L1 therapy strengthened an anti-tumor immune response and reduced tumor growth, while also easing pulmonary scarring in the animals. This combination therapy might be useful in treating lung…

The Pulmonary Fibrosis Foundation (PFF) is highlighting for smokers, and ex-smokers, the need to be aware of pulmonary fibrosis (PF) and their higher risk of developing this disease. It is well-established that tobacco smoke contains many chemicals harmful to health, and its use is linked to cancer, cardiovascular disease, and…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Calprotectin may serve as a blood biomarker for disease severity in patients with idiopathic pulmonary fibrosis (IPF), a new study indicates. Calprotectin blood levels are significantly higher in IPF patients and show a correlation with measures of lung function. The study, “Serum calprotectin as new biomarker for disease…

A set of rare, disease-causing variants in genes that help control both the body’s immune response and cell adhesion — the process by which cells form connections with other cells or their environment — were found to be more common in China in patients with idiopathic pulmonary fibrosis (IPF)…

At-home evaluation of lung function using a spirometry device is a “feasible and valid measure” for people with idiopathic pulmonary fibrosis (IPF) who have preserved lung function, clinical trial data show. However, further study is needed to evaluate the utility of at-home evaluations, since correlations between changes seen at…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…