News

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

Uplizna (inebilizumab-cdon) was just as safe and effective in people with neuromyelitis optica spectrum disorder (NMOSD) treated after only one symptomatic attack as those treated after two or more attacks, according to an analysis of N-MOmentum Phase 2/3 trial data. This sub-analysis had sought to understand how a…

The video series “Imagine My Life With NMO” has been launched by the Sumaira Foundation to help others in understanding what daily life can be like with neuromyelitis optica spectrum disorder (NMOSD). In the series, Sumaira Ahmed, the European foundation’s creator and a NMOSD patient, interviews other patients, their loved…

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

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…