News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

The therapeutic effects of Prograf (tacrolimus) in myasthenia gravis (MG) patients are influenced not only by dosage, but also by patients’ genetic profiles and other medications they may be taking for the disease, according to a study from China. The findings point…

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…