FDA approves first treatment for Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder

The U.S. Food and Drug Administration today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS.
Source: World Pharma News - Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news

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We reported a pediatric proband exhibiting muscle weakness in the trunk and limbs with skeletal malformation and intellectual disability and performed whole-exome sequencing (WES) of the proband parent-offspring trio. Results revealed a new compound heterozygous mutation in AGRN: c.125A>C (p.Glu42Ala) in the N-terminal agrin domain (NtA) and c.4516G>A (p.Ala1506Thr) in the laminin G1 domain (LG1). Bioinformatic analysis predicted the mutation as possibly pathogenic. The new compound heterozygous mutation in AGRN may disrupt agrin's known function of bridging laminin and α-dystroglycan and undermine the formatio...
Source: Frontiers in Neurology - Category: Neurology Source Type: research
We report the case of one patient who developed myositis and myasthenia during nivolumab treatment for metastatic lung squamous carcinoma. Moreover, we reviewed literature data in order to identify similar cases in cancer patients treated with immune-checkpoints inhibitors. A 65-year-old patient, who had previously received a first-line platinum-based therapy, developed diplopia and ptosis 4 weeks after the start of nivolumab. Although antibodies associated with myositis, myasthenia gravis and paraneoplastic syndromes were absent, immune-related myositis and myasthenia were diagnosed. Corticosteroids, immunoglobulin...
Source: Anti-Cancer Drugs - Category: Cancer & Oncology Tags: Case Reports Source Type: research
Autoimmune polyglandular syndrome type 2 (APS-2) is a rare and complex clinical entity, and little is known about its etiology and progression.
Source: BMC Endocrine Disorders - Category: Endocrinology Authors: Tags: Case report Source Type: research
Conclusion: The decreased mitochondrial membrane potential and mitophagy in Tregs in the MG group may be related to a decreased inhibition of Treg proliferation. The mitochondrial membrane potential was increased after adding the autophagy agent Rapa to enhance mitophagy, and the proliferation inhibition function of Tregs was also enhanced. The autophagy agent 3-MA down-regulated mitophagy, which decreased the mitochondrial membrane potential and inhibitory effect of Tregs. These results reveal the possible cellular immune mechanism of Treg dysfunction in MG.
Source: Frontiers in Neurology - Category: Neurology Source Type: research
Publication date: Available online 29 March 2020Source: Journal of Clinical NeuroscienceAuthor(s): Paulo José Lorenzoni, Cláudia Suemi Kamoi Kay, Raquel Cristina Arndt, Nyvia Milicio Coblinski Hrysay, Renata Dal-Pra Ducci, Otto H. Jesus Fustes, Ana Töpf, Hanns Lochmüller, Lineu Cesar Werneck, Rosana Herminia Scola
Source: Journal of Clinical Neuroscience - Category: Neuroscience Source Type: research
ConclusionsIn Japanese LEMS patients, the long ‐term neurological outcome is generally favorable. Our results also suggest that even in LEMS with SCLC patients, successful treatment for their cancer could result in sustained improvement of neurological symptoms.
Source: Clinical and Experimental Neuroimmunology - Category: Neurology Authors: Tags: ORIGINAL ARTICLE Source Type: research
With interest we read the article by Sugimoto et al. about a retrospective study on the “initial deterioration” of 51 patients with myasthenia gravis receiving 750–3000 mg/cycle intravenous methyl-prednisolone (IVMP) [1]. “Initial deterioration” was qualitatively and quantitatively defined, quantitatively as increase of two or more points on the myasthenia gravis activities of daily living (MG-ADL) score [1]. It was concluded that IVMP can be repeated after “initial deterioration” during a first cycle and that IVMP may lead to clinical improvement [1].
Source: Journal of the Neurological Sciences - Category: Neurology Authors: Tags: Letter to the Editor Source Type: research
CONCLUSION: The findings of this study indicates that late Masaoka-Koga staging and histology types are significantly associated with extended overall survival. Similarly, surgical resection and multimodality treatments play a significant role in thymic malignancies neoplasms therapy strategies to prolong survival rates. PMID: 32212790 [PubMed - in process]
Source: Asian Pacific Journal of Cancer Prevention - Category: Cancer & Oncology Tags: Asian Pac J Cancer Prev Source Type: research
Corona Virus Disease 2019 (COVID-19) is a new illness caused by a novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Symptoms are variable but typically include fever, cough, respiratory symptoms, diarrhea, reduction of smell and taste sensation. Severity ranges from mild to severe and the virus may lead to pneumonia, acute respiratory distress syndrome and death, in some patients. Nearly every country in the world has been affected by this virus and is currently defined as a pandemic, by the World Health Organization.
Source: Journal of the Neurological Sciences - Category: Neurology Authors: Tags: Clinical short communication Source Type: research
We report a case of pembrolizumab-induced myasthenia gravis that occurred in an 84-year-old Japanese female with metastatic urothelial carcinoma in multiple organs. She developed right ptosis 3  days after the second pembrolizumab treatment. Although prednisolone was administered, her symptoms did not change and dysphagia appeared. She needed the steroid pulse therapy for treatment eventually. On the other hand, 9 weeks after the first pembrolizumab treatment, reductions in the sizes of liver and adrenal metastases was observed. However, unfortunately, the severe immune-related adverse events did not allow her to...
Source: International Cancer Conference Journal - Category: Cancer & Oncology Source Type: research
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