News

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

Using robotic-assisted surgery to remove the thymus without putting a patient on an  immunosuppressant afterwards leads to higher myasthenia gravis (MG) improvement and remission rates, a study suggests. The study, “Independent long-term result of robotic thymectomy for myasthenia gravis, a single center experience” was published in the Journal of Thoracic…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

Ra Pharmaceuticals, which is now enrolling patients with generalized myasthenia gravis into a Phase 2 clinical trial, joined millions of health care advocates worldwide for the 11th annual Rare Disease Day, held to raise awareness and public understanding of rare diseases. The 2018 Rare Disease Day had as a…

People with refractory myasthenia gravis are at a higher risk of myasthenic crises and of being hospitalized compared to non-refractory patients, researchers report. Results reveal an increased disease burden in people with refractory myasthenia gravis, emphasizing the need for prevention and development of new treatment options for…

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

Researchers in Japan wrote about a new case study where treatment with the immunotherapy Opdivo (nivolumab) led to a relapse of myasthenia gravis (MG). The study is a new piece of evidence supporting previous concerns that this type of treatment may increase risk for autoimmune disorders. The…

In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…

University of Pennsylvania researchers used a component of rats’ muscle cell receptors to increase the punch of a myasthenia gravis treatment derived from an electric ray — an ocean animal that administers a shock like an electric eel. The team’s immune system suppression strategy alleviated the disease in rats and prevented…