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Subcutaneous Efgartigimod in Front of the FDA for Approval for MG

A novel human antibody fragment is under review by the US Food and Drug Administration (FDA) as a treatment for myasthenia gravis (MG) following the submission of a Biologics License Application, according to a recent press release from Argenx SE. “Every individual experiences generalized MG differently, which is why we’re excited about the possibility of…

Risk Factors Identified for Retinopathy in Sickle Cell Disease

Researchers reported that smoking and blood hemoglobin levels were among the modifiable risk factors for retinopathy related to sickle cell disease (SCD), according to a study published in the International Journal of Retina and Vitreous. Patients with SCD have sickle-shaped erythrocytes that often lead to painful vaso-occlusive crises, with sickle cell retinopathy (SCR) being one…

eyelid MG

NMD670 Receives FDA Orphan Drug Designation for Myasthenia Gravis

The US Food and Drug Administration (FDA) granted orphan drug designation to NMD670, a skeletal muscle-specific chloride ion channel (ClC-1) inhibitor designed to treat myasthenia gravis (MG), according to a recently published press release. Evidence collected from several animal models suggests that ClC-1 inhibition can increase the function of the neuromuscular union, thus improving muscle function…

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Systematic Review Aims to Identify Clinical Characteristics of Ocular MG

An upcoming systematic review aims to assess the clinical characteristics of ocular myasthenia gravis (MG) as well as the proportion of patients with ocular MG that progress to secondary generalized MG. Approximately half of the patients have positive antibodies against the neuromuscular junction receptors, acetylcholine receptors, and muscle-specific kinase receptors. However, the true proportion of…

Myasthenia gravis etiology

Routine Autoantibody Level Testing Not Recommended in MG

Although there appears to be a degree of correlation between autoantibody levels in patients with myasthenia gravis (MG) and disease severity, routine clinical use of autoantibody level testing is not recommended due to the limited evidence regarding the subject. The role of autoantibodies directed towards nicotinic acetylcholine receptors, autoantibodies muscle-specific kinase, and low-density lipoprotein receptor-related…

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New Trial Sets Out to Evaluate Mitoxantrone Hydrochloride in NMOSD

A new multicenter, randomized, double-blind, placebo-controlled phase 2 clinical trial aims to assess the safety and effectiveness of mitoxantrone hydrochloride liposome injection in patients with neuromyelitis optica spectrum disorder (NMOSD). The study will include 45 patients aged between 18 and 60 years with an NMOSD diagnosis made as determined by the 2015 International Consensus Diagnostic…

intravenous medication

Rituximab Trial Shows Decreased Symptoms and Need for Rescue Therapy in MG

Rituximab may be effective in preventing debilitating symptoms of myasthenia gravis (MG), indicating its efficacy as a potential first-line treatment, according to a randomized trial. The results showed that patients who received rituximab had fewer symptoms and needed less rescue therapy at week 16 compared to those in the placebo group. About 70% of patients…

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Single Low-Dose Rituximab Infusion May Improve Refractory MG Symptoms

The use of a single low-dose rituximab infusion may help improve symptoms in patients with refractory myasthenia gravis (MG), according to a study published in the Journal of Neurological Sciences. In the small study cohort, 13 out of the 17 patients responded to the rituximab treatment, with 6 even achieving asymptomatic status. The patients who…

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Novel Tool Used to Assess Cognitive Impairment Risk in Patients With NMOSD

Researchers from China developed a new classification scheme for cognitive impairments in patients with neuromyelitis optica spectrum disorder (NMOSD), a recent study suggests. Based on the scheme, the deterioration of motor ability in the arms and legs of the patients can potentially predict cognitive phenotypes in NMOSD. The findings were recently published in the journal…

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