News

Aeterna Zentaris GmbH has acquired exclusive global rights to develop, manufacture, and commercialize highly-specific, autoimmune modifying proteins for the treatment of neuromyelitis optica spectrum disorder (NMOSD). The company, a subsidiary of Aeterna Zentaris, reached an agreement with Julius-Maximilians-University Wuerzburg (JMU), in Germany, which developed the technology, called AIM Biologicals.

A branch of the European Medicines Agency has recommended that Enspryng (satralizumab) be approved to treat patients, 12 and older, with neuromyelitis optica spectrum disorder (NMOSD), who carry antibodies against the aquaporin-4 protein (AQP4-IgG). The recommendation, made by the agency’s Committee for Medicinal Products for Human Use…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

Soliris (eculizumab) significantly reduced the risk of disease relapses in people with neuromyelitis optica spectrum disorder (NMOSD) over nearly three years, according to new data from the clinical trial PREVENT and its extension study. Additional findings project that long-term Soliris treatment may reduce relapse risk over the course of…

Uplizna (inebilizumab-cdon) reduced pain among adults with neuromyelitis optica spectrum disorder (NMOSD), according to an analysis of the N-MOmentum Phase 2/3 clinical trial. The results were presented by Ho Jin Kim, MD, PhD, an investigator in the trial, at the 2021 virtual American Academy of Neurology Annual Meeting,…

Using a live cell-based assay to test for the presence of aquaporin-4 antibodies (AQP4-IgG) can accurately identify people who have neuromyelitis optica spectrum disorder (NMOSD), new research suggests. Scientists said the cell-based assay was “a highly specific diagnostic biomarker of NMOSD and did not yield false positive results.” These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

CorEvitas and the Guthy-Jackson Charitable Foundation (GJCF) announced plans to launch a registry study for people with neuromyelitis optica spectrum disorder (NMOSD). Called SPHERES — which stands for Synergy of Prospective Health & Experimental Research for Emerging Solutions in NMOSD — the observational study is expected to…

The first three months after giving birth are a particularly high-risk time for developing neuromyelitis optica spectrum disorder (NMOSD) and for experiencing relapses when the disease is already established, an expert emphasized in a recent webinar hosted by the The Sumaira Foundation for NMO. The pregnancy-focused webinar featured…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…