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Catalyst Pharmaceuticals recently enrolled the first patient into a Phase 3 clinical trial designed to evaluate the efficacy and safety of Firdapse (amifampridine phosphate) in patients with MuSK antibody-positive myasthenia gravis (MuSK MG). Firdapse is a neuronal potassium channel blocker that works to improve nerve impulses…

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…

Infection of the thymus by the virus that causes fifth disease — the human parvovirus B19 — can induce thymus overgrowth (hyperplasia) contributing to the development of myasthenia gravis, researchers have found. Reported in a study published in the journal Clinical Microbiology and Infection, this finding adds new…

A therapy that European regulators approved for treating Lambert-Eaton myasthenic syndrome (LEMS) can help manage myasthenia gravis symptoms, a study reports. The findings on Firdapse appeared in the Journal of Clinical Neuroscience. The title of the article is “3,4-Diaminopyridine for the treatment of myasthenia gravis with electrophysiological patterns…

Treatment with cannabinoids improved muscle function in a mouse model of myasthenia gravis (MG), suggesting they may have therapeutic potential for the disease, researchers say. The study, “Cannabinoid-induced increase of quantal size and enhanced neuromuscular transmission,” appeared in the journal Scientific Reports. Cannabinoids regulate multiple levels of neuronal communication,…

The European Commission has granted orphan drug status to argenx’s myasthenia gravis therapy ARGX-113 (efgartigimod), which is designed to rein in the overproduction of antibodies that damage junctions of nerve and muscle cells. U.S. regulators gave the therapy an orphan drug designation in October 2017. Autoimmune disorders like myasthenia…