Expanded use of the U.S. Food and Drug Administration-approved therapy Soliris (eculizumab) to treat myasthenia gravis (MG) could lead to a significant wholesale cost increase in 2018 and beyond, according to a new study by Prime Therapeutics. Prime manages pharmacy benefits for health plans, employers, and government programs, including Medicare and…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Treating late-onset myasthenia gravis (MG) with Rituxan (rituximab) eases disease symptoms, shows a positive safety profile, and allows patients to reduce other medications, researchers say. Their study, “B-cell depletion in late-onset myasthenia gravis is safe and effective; a case series (P6.439),” was presented as a poster at the…

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…

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…