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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

A rare case of myasthenia gravis (MG) complicated by a tumor in the thymus and an immunodeficiency known as Good syndrome has been documented by researchers, who warn against heavy use of certain immunosuppressants that are likely ineffective and pose a risk for serious infections. The…

Cyclophosphamide, a medicine with immunosuppressant abilities, may be a potential short-term alternative treatment for refractory — or difficult to treat — myasthenia gravis (MG), especially in lower-income countries, a small study from Mexico reports.   The study, “Intravenous cyclophosphamide monthly pulses in refractory myasthenia gravis,” was published in The…

People with refractory myasthenia gravis — those whose symptoms persist after treatment — are more likely to miss working hours and to be unemployed than those whose disease responds to treatment and are nonrefractory, a study found. The study, “Employment in refractory myasthenia gravis: A Myasthenia…

Cladribine, a medication for relapsing multiple sclerosis, eased symptoms and reduced the need for high-dosage corticosteroids in people with treatment-resistant myasthenia gravis, a small study shows. The study, titled “Cladribine in myasthenia gravis: a pilot open‐label study,” was published in the European Journal of Neurology.

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

Amneal has signed a licensing agreement with Kashiv BioSciences to develop and potentially commercialize K127, an extended-release tablet of pyridostigmine, for the treatment of myasthenia gravis (MG) in the United States. The deal offers Amneal exclusive rights over K127 development and marketing in the U.S. and the…

A first clinical trial of Descartes-08, a CAR T-cell therapy for people with generalized myasthenia gravis (GMG) by Cartesian Therapeutics, is enrolling patients. CAR T-cell therapies have shown promise in multiple cancer types, but this study is believed to be the first test of their safety and efficacy…

For people with thymomas, certain risk factors, such as clusters of developing B-cells, called germinal centers, in their thymuses, may make them more likely to develop myasthenia gravis (MG), a study suggests. Other factors that may contribute to MG development in people with thymomas include higher levels of…

Children with ocular myasthenia gravis respond well to treatment, as largely do children with generalized myasthenia gravis, according to a single-site study that found no “significant differences” in response rates. Still, half of its 22 ocular patients achieved remission, its researchers said. Children with ocular disease are also diagnosed at…