News

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

InnoCare Pharma will soon launch a Phase 2 clinical trial in China to test orelabrutinib, its oral investigational Bruton tyrosine kinase (BTK) inhibitor, to treat neuromyelitis optica spectrum disorder (NMOSD). The announcement comes after China’s National Medical Products Administration approved the company’s request to conduct such a trial,…

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…

This March, people with neuromyelitis optica spectrum disorder (NMOSD) are celebrating NMO Awareness Month by sharing stories of how the progressive autoimmune disorder has not stopped them from living meaningful lives. The initiative calls attention to NMOSD, a condition that affects approximately 15,000 people in the U.S. and 10,000…

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…

Blood levels of two proteins, GFAP and neurofilament light chain (NfL), may help predict the course of neuromyelitis optica spectrum disorder (NMOSD), according to new research. Findings also suggest that many NMOSD patients have evidence of brain lesions with active inflammation without any associated symptoms, which indicates that disease…

A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…

Enspryng (satralizumab) and tocilizumab — two interleukin-6 (IL-6) receptor inhibitors that prevent inflammation — are generally safe and effective for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a recent meta-analysis reported. Researchers determined these results by analyzing safety and efficacy outcomes across…

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…

It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.