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…
News
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…
At an educational seminar next month, experts will discuss advances in the diagnosis and care of neuromyelitis optica spectrum disorder (NMOSD), as well as the ongoing COVID-19 pandemic and how it is affecting the NMOSD community. Sponsored by the…
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Horizon Therapeutics is growing its rare disease portfolio by acquiring the biotechnology company Viela Bio, including the approved medicine Uplizna for treating neuromyelitis optica spectrum disorder (NMOSD). “This acquisition represents a significant step forward in advancing our strategy — to expand our pipeline,” Tim Walbert, chairman,…
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…
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