News

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…

Myasthenia gravis (MG) patients who undergo thymectomy — surgery to remove the thymus gland — have a reduced risk of pulmonary infection and a shorter stay in intensive care when taken off mechanical ventilation support, called extubation, within six hours after surgery, a study suggests. The study, “Early extubation…

People with myasthenia gravis can develop psychiatric symptoms, including depression and insomnia. However, recommendations regarding safe medication are insufficient. In this case study, clinicians suggest that alpha-blockers are a potential safe therapeutic option for relieving insomnia and anxiety in a female myasthenia gravis patient. The case report, “Management of…

Examining differences in the activity of certain genes may help explain why some patients with myasthenia gravis (MG) develop a difficult-to-treat form of the disease in the eyes. Researchers used laboratory models derived from patient cells to re-create MG muscle cells, and study their gene activity. The findings…

Placebo-treated myasthenia gravis (MG) patients experience as many adverse events as those receiving an active treatment during clinical trials, but this leads to few participant dropouts — and should not represent a problem for patient compliance to placebo during trials, a grouped analysis of studies shows. The study, “…

The presence of autoimmune diseases, such as myasthenia gravis (MG), should not be seen as a not be seen as a factor against treatment with immune checkpoint inhibitors in patients with advanced metastatic cancer, a case report says. The case report, “Metastatic Merkel cell carcinoma…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…