As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…
News
High pyridostigmine doses cause overactive bladder issues in people with myasthenia gravis (MG), according to a recent study. Because of this, doctors should take patients’ bladder-related discomfort into account when deciding treatment regimens, and consider prescribing lower pyridostigmine doses or alternative therapies. The study, “Overactive…
In two of three myasthenia gravis (MG) patients who contracted COVID-19, the use of immunosuppressants did not cause an exacerbation or myasthenic crisis, a case study reports, suggesting the treatments may be safe and beneficial for this group of patients. “Despite these…
Soliris (eculizumab) was found in a real-world setting to safely and effectively treat adults with generalized myasthenia gravis (gMG) who had stopped responding to other treatments, according to a post-marketing surveillance study in Japan. The study “Safety and effectiveness of eculizumab in Japanese patients with…
Myasthenia gravis (MG) can mimic the symptoms of a stroke and should not be ruled out even in younger patients, a recent medical case report suggests. The report, “Myasthenia gravis masquerading as acute stroke: a case report,” was published in the PanAfrican Medical Journal. MG…
When used over long periods of time, Soliris (eculizumab) does not increase the risk of infections in people with generalized myasthenia gravis (gMG), according to a new analysis of clinical trial data. Findings from this post hoc analysis were presented this week at the 2021 MDA Virtual Clinical…
Myasthenia gravis (MG) crisis, the disease’s most severe state, is associated with pronounced pro-inflammatory cellular and molecular changes, a recent study shows. Notably, several pro-inflammatory immune cells and molecules were found to be associated with symptom severity, adding to previous data showing a link between certain pro-inflammatory cells…
Rapid tapering of high-dose prednisone appears to be a feasible, safe, and effective option for people with moderate-to-severe generalized myasthenia gravis on a combination of corticosteroids and immunosuppressants, according to data from a Phase 4 trial. These findings were reported…
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
Ra Pharmaceuticals is enrolling adults with generalized myasthenia gravis (gMG) in RAISE, its global and pivotal Phase 3 trial evaluating the safety and efficacy of zilucoplan, the Muscular Dystrophy Association (MDA) has announced. The company started preparing to launch the RAISE (NCT04115293) study in early…
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