Coral Gables drug company under fire for drug pricing says patient care is 'top priority'

Coral Gables-based Catalyst Pharmaceuticals said patient care is its "top priority" after Sen. Bernie Sanders, I-Vermont, criticized the firm for its pricing of a medication that treats a rare autoimmune disorder. "Catalyst’s top priority is improving patient care in the [Lambert-Eaton Myasthenic Syndrome] community and potentially elsewhere within the neuromuscular community," Catalyst said in a statement provided to the South Florida Business Journal. "We will respond to Senator Sanders’…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news

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National Institutes of Health has awarded a research team from Yale and other universities $7.8 million to establish a rare disease network called MGNet.
Source: Yale Science and Health News - Category: Universities & Medical Training Source Type: news
The original version of this article unfortunately contained a mistake. Fifth sentence of the fourth paragraph in the section “Non-nAChR autoantibody targets in MG” should read as
Source: Journal of Neurology - Category: Neurology Source Type: research
Hospital admission trends in Myasthenia Gravis are largely unknown, so they were here investigated in Finland between 2004 and 2014 using national mandatory registry data. There were 2989 hospital admissions (59.7% for women) for 861 individuals (median 2 admissions/individual) The annual number of admissions (p  = .56), the age of admitted patients (p = .24) or length of stay (p = .20) showed no change during the study period. The proportion of infections as the primary diagnosis increased from 4.5% to 10.4% (p = .0056).
Source: Journal of the Neurological Sciences - Category: Neurology Authors: Source Type: research
Objectives: Thymic epithelial tumors (TETs) are rare malignant tumors that exhibit heterogeneous histology and clinical behavior. As immune check point inhibitors, drugs targeting anti-programmed cell death protein 1 (PD-1) and programmed death-ligand 1 (PD-L1) have shown remarkable results against many cancers; thus, the importance of PD-1/PD-L1 immunohistochemistry as a predictive or prognostic biomarker has grown. However, limited data on PD-L1 and PD-1 expression in TETs have been reported; moreover, these results have been variable. Here, we examined the expression of PD-1/PD-L1 proteins in TETs and analyzed the clini...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
Myasthenia Gravis (MG) – an autoimmune neuromuscular disease – is known by the production of autoantibodies against components of the neuromuscular junction mainly to the acetylcholine receptor, which cause the destruction and compromises the synaptic transmission. This disease is characterized by fluctuating and fati gable muscle weakness, becoming more intensive with activity, but with an improvement under resting. There are many therapeutic strategies used to alleviate MG symptoms, either by improving the transmission of the nerve impulse or by ameliorating autoimmune reactions with e.g.
Source: Journal of Neuroimmunology - Category: Allergy & Immunology Authors: Tags: Review Article Source Type: research
AbstractAryl hydrocarbon receptor (AhR), a type of transcriptional factor, is widely expressed in immune cells. The activation of AhR signaling pathway depends on its ligands, which exist in environment and can also be produced by metabolism. Normal expressions of AhR and AhR-mediated signaling may be essential for immune responses, and effects of AhR signaling on the development and function of innate and adaptive immune cells have also been revealed in previous studies. Recent studies also indicate that aberrant AhR signaling may be related to autoimmune diseases, including rheumatoid arthritis (RA), systemic lupus eryth...
Source: Inflammopharmacology - Category: Drugs & Pharmacology Source Type: research
Condition:   Myasthenia Gravis Intervention:   Drug: Rozanolixizumab Sponsor:   UCB Biopharma S.P.R.L. Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Consider slow-channel congenital myasthenic syndrome in the differential diagnosis of vocal cord paralysis.
Source: Neurology Clinical Practice - Category: Neurology Authors: Tags: Myasthenia Case Source Type: research
This case report highlights the importance of cholinesterase inhibitors management and neuromuscular block monitoring in the perioperative period for patients with myasthenia gravis.BMC Anesthesiology
Source: Medscape Today Headlines - Category: Consumer Health News Tags: Anesthesiology Journal Article Source Type: news
Myasthenia gravis (MG) is a neuromuscular autoimmune disease that affects the skeletal muscle through the production of autoantibodies against the nicotinic acetylcholine receptor at the neuromuscular junction, thus causing defective neuromuscular transmission in skeletal muscles [1]. MG occurs in 1: 7500 individuals, affecting women during the second to third decade and men in their fifth and sixth decade of life [2]. Typically, patients experience muscle weakness and fatigue, as well as fluctuating and fatigable extraocular (e.g.
Source: International Journal of Cardiology - Category: Cardiology Authors: Source Type: research
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