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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…

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…

Researchers have determined that video-assisted thoracic surgery to remove a thymoma — a tumor in the thymus — is a safe and effective surgical method. The study, “Video-assisted thoracic surgery for thymoma: long-term follow-up results and prognostic factors—single-center experience of 150 cases,” was published in the Journal of…

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…

Results of a Phase 1 clinical trial showed that the investigational therapy GTP-004 significantly reduced the side effects associated with the myasthenia gravis (MG) medication Mestinon (pyridostigmine). GT Biopharma, the therapy’s developer, plans to accelerate the development of GTP-004 by initiating a Phase 2 trial in myasthenia…

Myasthenia gravis patients appear to have lower levels of vitamin D than the general population, a study confirmed. The confirmation suggested that doctors need to monitor patients’ vitamin D levels to reduce the chance they will have additional autoimmune responses that can worsen their condition, the researchers said. Their study,…

Blood levels of a microRNA called miR-150-5p have been identified as a potential biomarker for AChR-positive myasthenia gravis, after surgical treatment was found to reduce its levels in a study. The study, “Thymectomy lowers the myasthenia gravis biomarker miR-150-5p,” was published in the journal Neurology…

Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half of the 7,000 rare diseases currently known — was the focus of a discussion by three top New York geneticists. The Feb. 28 conference,…