FDA Approves Ruzurgi for Children With Rare Autoimmune Disorder

TUESDAY, May 7, 2019 -- Ruzurgi (amifampridine) tablets are now approved to treat Lambert-Eaton myasthenic syndrome (LEMS) in children aged 6 to 17 years, the U.S. Food and Drug Administration announced yesterday. Ruzurgi is the first treatment to...
Source: Drugs.com - Pharma News - Category: Pharmaceuticals Source Type: news

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AbstractPurpose of ReviewImmune checkpoint inhibition for treatment of metastatic cancers is recognized to cause diverse immune-mediated neurological syndromes. This review will discuss current knowledge about the frequency and varied presentation of these syndromes affecting the peripheral nervous system as well as detail important diagnostic and management considerations.Recent FindingsImmune-related adverse events affect the peripheral nervous system more often than the central nervous system, and rates are likely underestimated. Most data regarding neurological immune –related adverse events are retrospective, an...
Source: Current Treatment Options in Neurology - Category: Neurology Source Type: research
Publication date: Available online 30 July 2020Source: Journal of Pediatric Surgery Case ReportsAuthor(s): F. Grasso, L. De Leonibus, M. Bertozzi, M. Sica, R. Angotti, L. Luzzi, F. Molinaro, M. Messina, P. Paladini
Source: Journal of Pediatric Surgery Case Reports - Category: Surgery Source Type: research
Background: Four main clinical phenotypes have been traditionally described in patients mutated in SCN4A, including sodium-channel myotonia (SCM), paramyotonia congenita (PMC), Hypokaliemic type II (HypoPP2), and Hyperkaliemic/Normokaliemic periodic paralysis (HyperPP/NormoPP); in addition, rare phenotypes associated with mutations in SCN4A are congenital myasthenic syndrome and congenital myopathy. However, only scarce data have been reported in literature on large patient cohorts including phenotypes characterized by myotonia and episodes of paralysis.Methods: We retrospectively investigated clinical and molecular featur...
Source: Frontiers in Neurology - Category: Neurology Source Type: research
The benefits of physical exercise for healthy individuals are well-established, particularly in relation to reducing the risks of chronic lifestyle related diseases. Furthermore, physical exercise has been seen to provide beneficial effects in many chronic diseases such as multiple sclerosis, rheumatoid arthritis, and chronic obstructive pulmonary disease and is therefore recommended as part of the treatment regimen. Myasthenia Gravis (MG) is a chronic autoimmune disease that causes neuromuscular transmission failure resulting in abnormal fatigable skeletal muscle weakness. In spite of this fluctuating skeletal muscle weak...
Source: Frontiers in Neurology - Category: Neurology Source Type: research
We present a 61 ‐year‐old man with severe myasthenia gravis, nonresponsive to conventional therapy. The patient was treated with individualized homeopathy, demonstrating significant improvement on his clinical status and no disease symptoms. AbstractWe present a 61 ‐year‐old man with severe myasthenia gravis, nonresponsive to conventional therapy. The patient was treated with individualized homeopathy, demonstrating significant improvement on his clinical status and no disease symptoms.
Source: Clinical Case Reports - Category: General Medicine Authors: Tags: CASE REPORT Source Type: research
Abstract Autoimmune neurological disorders, including neuromyelitis optica spectrum disorder, anti-N-methyl-D-aspartate receptor encephalitis, anti-MOG antibody-associated disorders, and myasthenia gravis, are clearly defined by the presence of autoantibodies against neurological antigens. Although these autoantibodies have been heavily studied for their biological activities, given the heterogeneity of polyclonal patient samples, the characteristics of a single antibody cannot be definitively assigned. This review details the findings of polyclonal serum and CSF studies and then explores the advances made by sing...
Source: Cellular and Molecular Immunology - Category: Molecular Biology Authors: Tags: Cell Mol Immunol Source Type: research
This article summarizes the milestones in the development of inebilizumab leading to this first approval for the treatment of AQP4-IgG seropositive NMOSD.
Source: Drugs - Category: Drugs & Pharmacology Source Type: research
CONCLUSIONS: This meta-analysis provides evidence that (1) β2-adrenergic receptor agonist therapy could be the first choice of pharmacological strategy for treating CMS with CHRNE mutations; (2) a single-drug-regime, rather than a combination therapy, should be the first choice of treatment; and (3) it is never too late to initiate pharmacological treatment. PMID: 32727330 [PubMed - as supplied by publisher]
Source: Current Neuropharmacology - Category: Drugs & Pharmacology Authors: Tags: Curr Neuropharmacol Source Type: research
Myasthenia gravis (MG) is an uncommon autoimmune neuromuscular junction disorder manifesting as fluctuating weakness of skeletal muscles. To add to its repertoire of mimicking a wide range of neurological disorders, the present case report is, to the best of our knowledge, the very first to describe MG masquerading as an idiopathic unilateral facial paralysis (Bell's palsy, BP). Our case report is distinct, highlights a novel clinical occurrence, offers new insights of how different neurological disorders may overlap with each other, and reminds neurologists to have a very broad and thorough comprehension for effective dia...
Source: Frontiers in Neurology - Category: Neurology Source Type: research
With interest we read the article by Delly et al. about a 56  years old female with myasthenia gravis (MG) since 5y for which she received pyridostigmine (240 mg/d), prednisone (40 mg/d), and intravenous immunoglobulins (IVIG, 650 mg/kg within 2d every 2 weeks), and with undetermined mixed connective tissue disease for which she received chloroquine (400 mg/d), who developed myasthenic crisis with respiratory insufficiency during pneumonia due to infection with SARS-CoV-2 [1]. Though the patient received vancomycin, cefepime, and azithromycin and prednisone was increased to 80 mg/d, she lastly had to be intub...
Source: Journal of the Neurological Sciences - Category: Neurology Authors: Tags: Letter to the Editor Source Type: research
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