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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…

Long-term treatment with Uplizna (inebilizumab-cdon) is safe and sustainably reduces the frequency of attacks in adults with neuromyelitis optica spectrum disorder (NMOSD), according to four-year data from the N-MOmentum Phase 2/3 clinical trial. Notably, these benefits also were observed among patients who previously received rituximab, an immunosuppressive therapy often…

Note: This story was updated March 29, 2021, to note that Uplizna is indicated for all NMOSD patients, regardless of the presence of aquaporin-4 water channel autoantibodies. Uplizna (inebilizumab-cdon) has been approved in Japan for preventing relapses in people with neuromyelitis…

Treatment with Soliris (eculizumab) does not increase the risk of infections or serious infections among people with neuromyelitis optica spectrum disorder (NMOSD) and antibodies against the protein aquaporin-4 (AQP4), even in those receiving accompanying immunosuppressive therapies. Those were the findings from a post hoc analysis of…

In summer 2014, 22-year-old Connor Judge was working as a landscaper when he experienced unusual pain in his legs and back. At first, the symptoms were presumed to be related to his landscaping work; visits to a few general practitioners were unremarkable. Then, on Aug. 29,…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

Activities are underway this month to raise awareness about neuromyelitis optica spectrum disorder (NMOSD), a rare progressive autoimmune disorder that affects about 15,000 people in the U.S. Patients, caregivers, and advocates nationwide are flooding social media platforms, garnering state proclamations, participating in educational webinars, and holding fundraisers — all…

An improved understanding of the biological processes that drive neuromyelitis optica spectrum disorder (NMOSD) has led to treatment advancements in recent years. In a presentation at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum 2021, Sean Pittock, MD, director of the Mayo Clinic’s Center for…

In most people with neuromyelitis optica spectrum disorder (NMOSD), autoantibodies against aquaporin-4 (AQP4) coordinate a complex series of immune activities that drive the disease. Better understanding of these disease-causing mechanisms may help in efforts to find new treatments for NMOSD patients. These mechanisms were explained in the lecture, “…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…