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.
Uplizna (inebilizumab-cdon) effectively reduced the severity of attacks in people with neuromyelitis optica spectrum disorder (NMOSD) who continued to experience attacks after being treated with the therapy during the N-MOmentum Phase 2/3 clinical trial, recent data analysis shows. The data also demonstrated lower levels of key disease-related biomarkers,…
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
People with neuromyelitis optica spectrum disorder (NMOSD) experience a high prevalence of co-existing conditions, or comorbidities, which are associated with much higher healthcare costs, a claims analysis demonstrated. In particular, pharmacy costs for NMOSD patients with any comorbidity were “significantly higher” than for those with no co-existing conditions: a…
Asymptomatic, or “silent,” MRI lesions are not common during disease remission in patients with aquaporin-4 antibody neuromyelitis optica spectrum disorder (AQP4-NMOSD) and myelin oligodendrocyte glycoprotein antibody disease (MOGAD), but are associated with imminent relapse when they do occur, according to a recent U.K. study. “Results of…
Rituximab may be a more effective immunosuppressive therapy for preventing a first relapse in people with neuromyelitis optica spectrum disorder (NMOSD) than mycophenolate mofetil (MMF), a new meta-analysis found. The anti-CD20 antibody rituximab, commonly used off-label in NMOSD, also appears to work better than azathioprine, though the results failed…
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