News

#RAREis Representation Program Promotes Equity, Diversity

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

Rare Disease Day Panel Opens Window to Patient Experience

BioNews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…

Study Links GI Symptoms to Low Muscle Mass in SSc

Low muscle mass is associated with gastrointestinal (GI) bloating in patients with systemic sclerosis (SSc), a recent study revealed. Disease duration, severity, and activity, as well as skin thickening, also were higher in patients with low muscle mass. “At the best of our knowledge, this is the first study…

Q&A With RARE-X Disease Data Platform Founder, Nicole Boice

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

Cytokine Profiles Identify Risk of PAH in SSc

Levels of signaling molecules called cytokines can be used to identify people with systemic scleroderma (SSc) who are at highest risk of developing abnormally high blood pressure in their lungs, a new study reports. The study, “Cytokine signatures differentiate systemic sclerosis patients at high versus low…