FDA approves new drug developed by Coral Gables company

The U.S. Food and Drug Administration has approved Firdapse, a new medicine developed by Coral Gables-based Catalyst Pharmaceuticals. The biopharmaceutical company (Nasdaq: CPRX) expects the drug to be commercially available in the first quarter of 2019. It was designed to treat Lambert-Eaton Myasthenic Syndrome, a rare autoimmune disease that causes muscle weakness and fatigue. "The approval of Firdapse is a crowning achieveme nt for our company and an important step forward in our transition into…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news

Related Links:

Myasthenia gravis (MG) with antibodies to the muscle-specific receptor tyrosine kinase (MuSK) is a distinct sub-group of MG, affecting 5–8% of all MG patients. MuSK, a receptor tyrosine kinase, is expressed at the neuromuscular junctions (NMJs) from the earliest stages of synaptogenesis and plays a crucial role in the development and maintenance of the NMJ. MuSK-MG patients are more severely affected and more refractory to treatments currently used for MG. Most patients require long-term immunosuppression, stressing the need for improved treatments. Ideally, preferred treatments should specifically delete the antigen...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Toll-like receptor (TLR)-mediated innate immune responses are critically involved in the pathogenesis of myasthenia gravis (MG), an autoimmune disorder affecting neuromuscular junction mainly mediated by antiacetylcholine receptor antibodies. Considerable evidence indicate that uncontrolled TLR activation and chronic inflammation significantly contribute to hyperplastic changes and germinal center (GC) formation in the MG thymus, ultimately leading to autoantibody production and autoimmunity. miR-146a is a key modulator of innate immunity, whose dysregulation has been associated with autoimmune diseases. It acts as inhibit...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Myasthenia gravis (MG) is an autoimmune disease caused by antibodies which attack receptors at the neuromuscular junction. One of the main difficulties in predicting the clinical course of MG is the heterogeneity of the disease, where disease progression differs greatly depending on the subgroup that the patient is classified into. MG subgroups are classified according to: age of onset [early-onset MG (EOMG; onset ≤ 50 years) versus late-onset MG (LOMG; onset> 50 years]; the presence of a thymoma (thymoma-associated MG); antibody subtype [acetylcholine receptor antibody seropositive (AChR+) and muscle-specific tyrosi...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Conclusion: The combination of physician-evaluated and patient-reported outcome measures provided a more discerning picture of patient status and response to treatment. Incorporating MG outcome measures into clinical practice would aid in modulating therapies.
Source: Annals of Indian Academy of Neurology - Category: Neurology Authors: Source Type: research
In conclusion, OPN likely exacerbates the pathogenesis of EAMG by promoting the differentiation of Th1 cells and inhibiting the differentiation of Treg cells. PMID: 32109680 [PubMed - as supplied by publisher]
Source: International Immunopharmacology - Category: Allergy & Immunology Authors: Tags: Int Immunopharmacol Source Type: research
Myasthenia Gravis (MG) is an autoimmune disease of the neuromuscular junction (NMJ), with autoantibodies binding proteins in the NMJ detected in ∼85% of the patients. However, 10-15% of MG patients present with no known antibodies [1,2]. Congenital myasthenic syndromes (CMS), in turn, are disorders of the NMJ due to various genetic defects in components essential for NMJ structure and function. Symptoms in CMS patients typically start shor tly after birth or during childhood, with an adult onset being rarer.
Source: Neuromuscular Disorders - Category: Neurology Authors: Tags: Case report Source Type: research
In conclusion, our study summarizes the regulatory transcription factors that drive expression of AChR+ and MuSK+ MG-associated miRNAs. Our findings of elevated miR-21-5p and miR-30e-5p expression in immune cells upon inflammatory stimulation and the suppressive effect of corticosteroids strengthens the putative role of these miRNAs in the MG autoimmune response.
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Discussion Myasthenia gravis (MG) is a problem of the neuromuscular junction which causes muscle weakness. It can occur in all ages and have a range of symptoms from mild localized disease to mortality-threatening respiratory failure. MG occurs in 1.7-30 cases/million, with a prevalence of 77.7 cases/million. Pediatric patients comprise 10-15% of all patients with MG. In various Asian populations, the juvenile MG can be up to 50% of all of the MG cases. Fluctuations in muscle weakness is a hallmark of the disease. As a reminder, “[i]n normal synaptic transmission in the neuromuscular junction, the axon is depolarize...
Source: PediatricEducation.org - Category: Pediatrics Authors: Tags: Uncategorized Source Type: news
CONCLUSIONS These results indicate that grilled nux vomica ameliorates EAMG by depressing the TLR-4/NF-kappaB signaling pathway, and hub genes may serve as potential targets for MG treatment. PMID: 32052794 [PubMed - in process]
Source: Medical Science Monitor - Category: Research Tags: Med Sci Monit Source Type: research
AbstractLambert ‐Eaton myasthenic syndrome (LEMS) is an autoimmune neuromuscular junction disease associated with P/Q‐type voltage‐gated calcium channels (P/Q‐type VGCCs) autoantibodies1. P/Q ‐type VGCCs present at the presynaptic motor nerve terminals and their antibodies induce a reduction in neurotransmitter release, leading to the characteristic muscle weakness associated with the disease. Approximately half of LEMS patients have small‐cell lung carcinoma (SCLC) that occurs as a paraneoplastic disorder.
Source: Clinical and Experimental Neuroimmunology - Category: Neurology Authors: Tags: NEW DEVELOPMENTS IN NEUROIMMUNOLOGY Source Type: research
More News: Autoimmune Disease | Biotechnology | Food and Drug Administration (FDA) | Health Management | Myasthenia Gravis