Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
News
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…
Patients with mild myasthenia gravis (MG) but with poorer nerve cell response in initial diagnostic tests are more likely to progress to more aggressive therapy regimens than those with better nerve responses, known as decrement scores. The study “Baseline Decrement in Patients with Mild Myasthenia Gravis Predicts Immunomodulation…
Myasthenia gravis (MG) can co-occur with premature ovarian failure (POF). Prompt and proper MG treatment, however, can help restore reproductive organ health, a recent report of two cases suggests. The case report, titled “Clinical characteristics and relationship between myasthenia gravis and premature ovarian failure: report of two…
High levels of PCO2 — a measure of the amount of carbon dioxide gas dissolved in blood — may be a useful biomarker for higher mortality risk in people with myasthenia gravis undergoing acute respiratory crisis, a study suggests. The study, titled “Myasthenic crisis treated in a…
The use of blood cholesterol-lowering medications called statins is linked to a small risk of myasthenia gravis (MG), according to a large World Health Organization database study. The study, “Statin‐induced myasthenia: a disproportionality analysis of the WHO’s VigiBase pharmacovigilance database,” was published in the journal Muscle&Nerve. Statins…
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…
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…
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