News

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

After its approval last year, Cimzia (certolizumab pegol) may now be the most prescribed therapy in the U.S. for people with non-radiographic axial spondyloarthritis (nr-axSpA), according to an online survey of 98 rheumatologists. Cimzia is the only therapy currently approved in the U.S. for the treatment of…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

Lower blood levels of retinol-binding protein 4 (RBP4) — a protein associated with inflammation, among other processes — in people with ankylosing spondylitis (AS) are associated with better treatment responses to Humira (adalimumab), a study shows. These findings suggest that RBP4 may be used as a biomarker to…

Both treatment with Humira (adalimumab) and smoking can alter the composition of the gut microbiome — the microorganisms living in the gut — in people with ankylosing spondylitis (AS), a new study shows. Results also revealed that nonsmokers showed greater reductions in disease activity than smokers upon starting Humira. The…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

Elevated levels of a protein produced in both bones and the gut, known as lipocalin 2 (LCN2), could explain the overlap between ankylosing spondylitis (AS) and inflammatory bowel disease (IBD), a new study suggests. Its researchers also suggested that therapies aimed at lowering LCN2 levels may help…