Cabaletta Bio has been cleared by the U.S. Food and Drug Administration (FDA) to start first-in-human clinical testing of MuSK-CAART, a cell-based therapy being developed to improve muscle strength in people with MuSK antibody-positive myasthenia gravis and help them in their activities of daily living. “We look forward to…
Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A Window…
To celebrate Rare Disease Day, on Feb. 28, Janssen has launched a refreshed national campaign, called iMaGineMyMG, to increase awareness about myasthenia gravis (MG), provide support to people living with the disease and their families, and open up opportunities for potential research. The company has redesigned the campaign’s…
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 burden,…
Researchers report having uncovered the atomic structure of the acetylcholine receptor, the target of the abnormal immune response that causes most cases of myasthenia gravis (MG). They also detailed how the receptor is activated by changes in its shape when combined with two small molecules. Their work could help…
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…
Nearly half of all people with myasthenia gravis (MG) in Poland were admitted to a hospital a mean of two times in 2018, although not always for complications of their disease, a recent nationwide database study reported. Reasons for hospital stays included relevant treatments,…
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.
Patients with well-controlled myasthenia gravis (MG) and no co-existing conditions, or comorbidities, are more likely to have milder COVID-19 and experience more favorable outcomes, according to a recent study. “Our study has shown that COVID-19 does not necessarily have a severe course or poor outcome in MG patients. Absence…
Lower levels of antibodies targeting acetylcholine receptors in the bloodstream of people with myasthenia gravis (MG) were associated with improved clinical status, a study demonstrated. These findings showed not only that antibody blood tests are useful for diagnosing MG, but also that repetitive testing may be “valuable as a…
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