News

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…

A lesser known variety of myasthenia gravis (MG), called double-seropositive, appears to be a subtype of MuSK-antibody positive MG, a single-site study in China reports. Recognizing this subtype could help in properly diagnosing patients, allowing for better treatment outcomes, its scientists said. Their study, “AChRAb and…

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…

In a $3.05 billion deal, Horizon Therapeutics will acquire Viela Bio, the developer of inebilizumab, a potential treatment for autoimmune diseases such as myasthenia gravis (MG), the two companies announced. Horizon plans to advance inebilizumab, already approved for treating another rare autoimmune disease, as a therapy for decreasing disease activity…

High levels of autoantibodies against acetylcholine receptors (AChR) in the blood of people with thymoma — a tumor of the thymus — can help to identify patients at risk of developing myasthenia gravis (MG) after tumor surgery, a study suggests. Larger tumors, and partial tumor resection might also increase this risk.

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

The National Organization for Rare Disorders (NORD), a nonprofit organization dedicated to supporting those with rare diseases, has launched new patient assistance programs for people with myasthenia gravis (MG) living in the U.S. The new assistance programs aim to help patients who have limited financial resources with…