- |||||||||| methylprednisolone / generics, doxorubicin hydrochloride / generics
Clinical, Retrospective data, Journal: Multimodality therapy for thymoma patients with pleural dissemination. (Pubmed Central) - Aug 8, 2019
Multidisciplinary treatment using induction CAMP therapy and surgical resection for thymoma patients with pleural dissemination was effective and feasible. Because of the low recurrent rate of disease, young patients with good cardiopulmonary function and well-controlled MG might be good candidates for EPP.
- |||||||||| Enrollment closed, Enrollment change, HEOR: Mya-BoD: Burden of Disease in Myasthenia Gravis (clinicaltrials.gov) - Aug 8, 2019
P=N/A, N=1680, Active, not recruiting,
Because of the low recurrent rate of disease, young patients with good cardiopulmonary function and well-controlled MG might be good candidates for EPP. Recruiting --> Active, not recruiting | N=1200 --> 1680
- |||||||||| Journal: Fc Fusion Protein as a Novel Treatment for Myasthenia Gravis (Pubmed Central) - Aug 8, 2019
AChR-Fc exhibited dual activities: it neutralized anti-AChR antibodies and demonstrated an enhanced cytotoxicity against autoantibody-producing B cells. Therefore, AChR-Fc is a novel biomolecule that can be used in the treatment of patients with MG, and potentially overcomes the disorder.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Eculizumab Treatment for Refractory Generalized Myasthenia Gravis (Pubmed Central) - Aug 8, 2019
Recently, complement inhibitor, the humanized monoclonal anti-C5 antibody eculizumab, complement inhibitor, was approved for patients with anti-AChR antibody-positive generalized refractory MG in Japan. In this review, we focus on the role of complements in the pathogenesis of MG and the action mechanism, efficacy, and future prospects of eculizumab.
- |||||||||| methylprednisolone / generics
Intestinal Pseudo-Obstruction Associated With Seropositive Myasthenia Gravis, Thymoma, and Positive CV-2 Antibodies (Exhibit Halls 3 and 4 (Street Level)) - Aug 8, 2019 - Abstract #ACG2019ACG_3089;
The patient was treated with IV methylprednisolone and pyridostigmine with marked improvement in symptoms...Anti-CV2 antibody was positive, which was reported in only one previously published case. In summary, patients with thymomas or MG should have testing for paraneoplastic antibodies; they tend to respond to treatment with immunosuppression and acetylcholinesterase inhibitors.
- |||||||||| Brunner's Gland Hyperplasia: A Massive Duodenal Lesion (Exhibit Halls 3 and 4 (Street Level)) - Aug 8, 2019 - Abstract #ACG2019ACG_3087;
Few cases reported in literature have been managed with laparotomy. The potential cause of our patient’s large Brunner’s gland mass could be gastric acid hypersecretion as he had severe GERD with peptic esophageal strictures.
- |||||||||| Malakoplakia of the Rectum Masquerading as a Tumor: A Case Report (Exhibit Halls 3 and 4 (Street Level)) - Aug 8, 2019 - Abstract #ACG2019ACG_2478;
Pathologists should be aware that malakoplakia can coexist with malignancy and tuberculosis, therefore, thorough sampling of mass lesions and appropriate adjunctive stains are advisable. Risk factors in our case included an immunocompromised state, multiple concurrent infections, and advanced malignancy, although no malignancy was identified in the biopsies.
- |||||||||| Remicade (infliximab) / Mitsubishi Tanabe, J&J, Opdivo (nivolumab) / Ono Pharma, BMS, Yervoy (ipilimumab) / Ono Pharma, BMS
Immunotherapy-Mediated Hemorrhagic Gastritis After Ipilimumab and Nivolumab Therapy (Exhibit Halls 3 and 4 (Street Level)) - Aug 8, 2019 - Abstract #ACG2019ACG_2259;
Physicians should be aware that checkpoint inhibitor-induced toxicity of the upper GI tract can occur early after initiation of therapy and have a severe, refractory course. Management includes exclusion of infectious process and immunosuppressive therapy.
- |||||||||| Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
Trial completion date: Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG (clinicaltrials.gov) - Aug 8, 2019
P3, N=60, Recruiting,
Management includes exclusion of infectious process and immunosuppressive therapy. Trial completion date: Sep 2019 --> Dec 2019
- |||||||||| Review, Journal: Neuronal Antibodies and Associated Syndromes. (Pubmed Central) - Aug 6, 2019
Existing research provides evidence revealing a spectrum of antibodies linked to the development and progression of neurological diseases. However, further antibody testing and studies should be performed to validate the relationship between conditions and antibodies.
- |||||||||| Journal: Homeostatic synaptic plasticity at the neuromuscular junction in myasthenia gravis. (Pubmed Central) - Aug 1, 2019
The homeostatic changes occurring in myasthenia gravis appear to involve the presynaptic nerve terminal and may even involve changes in the excitability of motor neurons within the spinal cord. The finding of presynaptic homeostatic synaptic plasticity in myasthenia gravis leads us to propose that changes in the motor unit in myasthenia gravis may be more widespread than previously appreciated.
- |||||||||| Journal: A unique subphenotype of myasthenia gravis. (Pubmed Central) - Aug 1, 2019
A genetic study, using extended whole-exome sequencing and an "extreme" phenotype sample of OP-MG versus control MG cases differing only by their EOM responsivity to therapy, discovered several potentially functional OP-MG risk variants. These variants implicate myogenesis and gangliosphingolipid biosynthesis pathways at the EOM endplates in OP-MG.
- |||||||||| Journal: Learning from the past: reflections on recently completed myasthenia gravis trials. (Pubmed Central) - Aug 1, 2019
We illustrate how other design characteristics (e.g., treatment duration, strategy for steroid tapering, selection of the primary outcome, and principal analytic approach) may affect the sensitivity of a trial to demonstrate therapeutic effects. Finally, we consider the importance of placebo effects, being careful to differentiate these from therapeutic effects observed in the placebo group, and discuss how the use of combined outcome measures may minimize placebo effects.
- |||||||||| Biomarker, Review, Journal: Diagnostic utility of cortactin antibodies in myasthenia gravis. (Pubmed Central) - Aug 1, 2019
Patients with dSNMG cortactin(+) MG presented ocular or mild generalized MG without bulbar symptoms. We conclude that cortactin autoantibodies are biomarkers of MG that, when present, suggest that the disease will be mild.
- |||||||||| Review, Journal: Neuromuscular synapse electrophysiology in myasthenia gravis animal models. (Pubmed Central) - Aug 1, 2019
With intracellular microelectrode measurement of (miniature) endplate potentials at NMJs in ex vivo nerve-muscle preparations from MG animal models, these functional synaptic defects have been determined in much detail. Here, we describe the electrophysiological events at the normal NMJ and the pathoelectrophysiology at NMJs of animal models for MG.
- |||||||||| Biomarker, Clinical, Review, Journal: Circulating microRNAs as potential biomarkers in myasthenia gravis patients. (Pubmed Central) - Aug 1, 2019
In MuSK(+) MG, another profile of circulating miRNAs was found, including upregulation of the let-7 family of miRNAs. Here, we summarize the potential role of circulating miRNAs as biomarkers in general and in MG, and highlight important considerations for the analysis of circulating miRNA.
- |||||||||| Review, Journal: Thymus involvement in early-onset myasthenia gravis. (Pubmed Central) - Aug 1, 2019
Interferon (IFN)-β is overexpressed in MG thymus and could orchestrate thymic changes associated with MG. Knowledge about how IFN-β is induced in MG thymus and why its expression is sustained even long after disease onset would be of interest in the future to better understand the etiological and physiopathological mechanisms involved in autoimmune MG.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Myasthenia gravis: the role of complement at the neuromuscular junction. (Pubmed Central) - Aug 1, 2019
Eculizumab treatment improved symptoms compared with placebo in a phase II study in patients with refractory gMG. Direct complement inhibition could preserve NMJ physiology and muscle function in patients with anti-AChR+ gMG.
- |||||||||| Rituxan (rituximab) / Roche, Biogen
Review, Journal: Myasthenia gravis with antibodies to MuSK: an update. (Pubmed Central) - Aug 1, 2019
In uncontrolled studies, B cell depletion with rituximab proved effective in most patients with refractory disease, inducing prolonged clinical responses associated with a sustained reduction of serum antibody levels. Promising results from experimental studies and case reports suggest that both 3,4-diaminopyridine and albuterol may be effective as symptomatic agents.
- |||||||||| Clinical, Review, Journal: Clinical and research strategies for limb-girdle congenital myasthenic syndromes. (Pubmed Central) - Aug 1, 2019
Tailored animal and cell models are essential to elucidate the exact function and pathomechanisms at the neuromuscular synapse that underlie LG-CMS. The integration of genomics and proteomics data derived from these models and patients reveals new and often unexpected insights that are relevant beyond the rare genetic disorder of LG-CMS and may extend to the functioning of mammalian synapses in health and disease more generally.
- |||||||||| Review, Journal: Mechanisms underlying B cell immune dysregulation and autoantibody production in MuSK myasthenia gravis. (Pubmed Central) - Aug 1, 2019
Third, using complementary approaches, including production of human monoclonal antibodies, we determined that circulating plasmablasts directly contribute to the production of MuSK-specific autoantibodies in patients experiencing relapse following B cell depletion therapy. These collective findings contribute to defining a mechanistic model that describes MuSK MG immunopathogenesis.
- |||||||||| fluoxetine / generics
Review, Journal: Therapeutic strategies for congenital myasthenic syndromes. (Pubmed Central) - Aug 1, 2019
...The present-day drug repertoire comprises acetylcholinesterase inhibitors (mainly pyridostigmine), 3,4-diaminopyridine (3,4-DAP), ephedrine, salbutamol/albuterol, open-channel blockers (fluoxetine, quinidine), or a combination of these...Here, we summarize for the clinician the therapeutic strategies employed in this ever-evolving disease spectrum. We also address the barriers to treatment and discuss the treatment of CMS in pregnancy.
- |||||||||| Review, Journal: Developing treatment guidelines for myasthenia gravis. (Pubmed Central) - Aug 1, 2019
The executive summary of the guidance statement was published with open access to facilitate access by patients and healthcare professionals, and the full statement, with extensive background information, is available online. The guidance statement is a living document that will require updates as new treatments and new information on current treatments become available.
- |||||||||| Clinical, Journal, HEOR: Patients With Migraine Have Substantial Reductions in Measures of Visual Quality of Life. (Pubmed Central) - Aug 1, 2019
In fact, patients with chronic migraine may have visual quality of life impacts that are as significant as those associated with other common neuro-ophthalmic disorders. Future studies of the overall disease burden in patients with migraine should include an evaluation of the effects on visual functioning.
- |||||||||| Journal: Structures of DPAGT1 Explain Glycosylation Disease Mechanisms and Advance TB Antibiotic Design. (Pubmed Central) - Aug 1, 2019
Our structures of DPAGT1 with the substrate UDP-GlcNAc and tunicamycin reveal substrate binding modes, suggest a mechanism of catalysis, provide an understanding of how mutations modulate activity (thus causing disease) and allow design of non-toxic "lipid-altered" tunicamycins. The structure-tuned activity of these analogues against several bacterial targets allowed the design of potent antibiotics for Mycobacterium tuberculosis, enabling treatment in vitro, in cellulo and in vivo, providing a promising new class of antimicrobial drug.
- |||||||||| Journal: Critical Care of Neuromuscular Disorders. (Pubmed Central) - Aug 1, 2019
Developing an airway management protocol could have implications on outcome and length of stay for patients with neuromuscular disorders in the ICU. Tending to the appropriate nuances of each patient who is critically ill with a neuromuscular disorder through evidence-based medicine can also have implications on length of stay and outcome.
- |||||||||| Clinical, Journal: Non-uremic calciphylaxis in a patient with multiple rheumatologic diseases. (Pubmed Central) - Aug 1, 2019
Herein we present a patient with calciphylaxis who had normal kidney function and numerous rheumatologic diseases, namely systemic lupus erythematosus (SLE), Sjogren syndrome (SS), and myasthenia gravis (MG). We review the pathophysiology, possible mechanisms, and management for non-uremic calciphylaxis.
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