News

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

An under-the-skin injection formulation of efgartigimod is as effective as the therapy’s infusion version, sold as Vyvgart (efgartigimod alfa-fcab), at lowering immunoglobulin G antibody levels in people with generalized myasthenia gravis (gMG), according to top-line data from the ADAPT-SC trial. Based on these findings, Argenx, the therapy’s developer, plans…

This year’s MGNet Scholar award, funded by the Myasthenia Gravis Rare Disease Network (MGNet), has gone to a scientist at Yale University who is investigating the immune mechanisms behind the development of myasthenia gravis (MG) in people who have been treated with a type of immunotherapy. The awardee…

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

Soliris (eculizumab) is better than rituximab at controlling myasthenia gravis (MG) symptoms over a two-year period, according to a new analysis. Results were detailed in the study, “Eculizumab versus rituximab in generalised myasthenia gravis,” published in the Journal of Neurology, Neurosurgery and Psychiatry. Soliris…

Immunosuppressant medications significantly reduced the risk of ocular myasthenia gravis (MG), which affects only eye and eyelid muscles, progressing to a generalized form of the disease that affects other muscles, a Chinese study suggests. These findings indicate that immunosuppressant medications should be considered early on in the treatment…

Software maker BrightInsight is teaming up with the biopharmaceutical company UCB to develop and commercialize a new mobile app to help people with myasthenia gravis (MG) manage their disease and improve their care and clinical outcomes. “MG is a rare, challenging and unpredictable disease, and innovation is…

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

Men with late-onset myasthenia gravis (MG) show poorer memory and spatial orientation, as well as lower-than-normal volumes of brain regions involved in such functions, than their healthy peers, according to a small single-center study in Germany. These findings add to a number of studies reporting cognitive deficits in MG…

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…