News

Camurus AB and Ra Pharmaceuticals have signed an agreement to develop and manufacture a long-acting injectable formulation of zilucoplan for patients with complement-mediated disorders, such as generalized myasthenia gravis. Using Camurus’s proprietary FluidCrystal technology, the subcutaneously injected formulation will allow patients with complement-mediated disorders — such as…

People with myastenia gravis (MG) whose disease is refractory, or resistant, to conventional therapies, are more likely to have disease flare-ups, visit an emergency room, be hospitalized, or be admitted to an intensive care unit — using more healthcare resources — than patients with nonresistant MG, a U.S. study…

Muscle cells of myasthenia gravis patients produce large amounts of the programmed cell death ligand 1 (PD-L1) — a substance produced by different types of cells to prevent the overactivation of immune cells — which may affect how these patients’ immune systems work, a study says. The findings of…

Myasthenia gravis and multiple sclerosis (MS) may share immunological features, according to researchers who studied the rare case of a man who developed both illnesses. The case was described in the study “Co-occurrence of multiple sclerosis and myasthenia gravis: A case report and review of…

It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…

In certain people whose myasthenia gravis (MG) has been in stable remission for a long time, using the immunotherapy Opdivo (nivolumab) to treat a relapsed lymphoma may be possible, researchers in Germany report. Their case study differs from others that linked treatment with immunotherapies like Opdivo to a higher risk of…

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

Treatment with synthetic RNA molecules targeting the C5 complement system protein in the liver may be effective in people with myasthenia gravis (MG), according to a new study in rats, mice and monkeys. The findings also indicate that marked benefits may be achieved without frequent dosage. The study, “…

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…