Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…
News
Zai Lab’s application seeking approval of efgartigimod in China to treat adults with generalized myasthenia gravis (gMG) has been accepted by the country’s regulatory agency, the National Medical Products Administration (NMPA). “We are pleased to have the NMPA’s acceptance of the [application] filing for efgartigimod alfa injection…
A man developed signs of myasthenia gravis (MG) with eye symptoms about a month after receiving the first shot of Oxford-AstraZeneca’s COVID-19 vaccine, a case study reports. “This case report implies that COVID-19 vaccination may cause myasthenia gravis with ocular symptoms,” the researchers wrote. “But we should also consider…
Generalized myasthenia gravis (gMG) may be associated with a deficiency in a type of immune cell called myeloid-derived suppressor cells (MDSCs), a study suggests, and that restoring their function and increasing their numbers may help reduce symptoms. Treatment with immunosuppressants resulted in an expansion of MDSCs, which was linked…
The U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on Phase 3 trials that are testing Sanofi’s experimental BTK inhibitor tolebrutinib. The move affects a trial in people with myasthenia gravis (MG), as well as several studies evaluating tolebrutinib for different…
Contrary to increasing evidence, high-intensity inpatient rehabilitation was not found, in a Japanese study, to speed the recovery of people with myasthenia gravis (MG) who were intubated after a myasthenic crisis. Instead, according to researchers, “high-intensity rehabilitation was associated with delayed recovery in the ADL [activities of daily living],”…
European regulators have recommended efgartigimod be approved as an add-on therapy for adults with generalized myasthenia gravis (gMG) who have anti-acetylcholine receptor (AChR) antibodies. This recommendation, from the Committee for Medicinal Products for Human Use (CHMP), will serve as the basis for a final regulatory decision from the…
Soliris (eculizumab) safely and rapidly led to clinical benefits in people with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody positive, according to data from a small real-world U.S. study. Moreover, most patients discontinued maintenance immunomodulatory treatment. The study, “Safety and outcomes of eculizumab for acetylcholine…
Two molecules in blood cells may be potential biomarkers for diagnosing myasthenia gravis (MG), a study suggests. Researchers found levels to be lower in mononuclear cells — a mixture of different cell types that circulate in the bloodstream and have a round nucleus — of patients with MG than…
Two doses of the COVID-19 vaccine did not worsen symptoms in 200 people with myasthenia gravis (MG), with mostly injection site-related side-effects reported, matching those seen in the general population, a study reported. These findings contrast with those of a recent study suggesting that, in very rare cases,…
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