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…
News
Vaccination does not trigger autoimmunity linked to myasthenia gravis in pediatric patients, a study based on records of the U.S. Department of Health and Human Services suggests. The findings of the study “Pediatric Myasthenia Gravis After Vaccination in the United States. A Report from the CDC/FDA Vaccine…
Ra Pharma’s RA101495, a potential treatment for generalized myasthenia gravis patients, showed preliminary signs of halting red blood cell destruction in healthy volunteers, results of an early clinical trial show — justifying the Phase 2 trial in patients now underway. Data reporting on the Phase 1 trial results — showing that RA101495…
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…
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 gravis…
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