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- || Interesting. So the effect is coming from the muscle (like myasthenia gravis), not the motor neuron. And estrogen protects AChRs from this effect? I wonder if the girls catch up with the boys post-OVX. (Twitter) - Apr 16, 2020
- | Journal: Takotsubo cardiomyopathy, or 'broken-heart syndrome', with concomitant myasthenic crisis. (Pubmed Central) - Apr 15, 2020
No abstract available
- || Clinical, Journal: The utility of a single simple question in the evaluation of patients with non-diabetic polyneuropathy. (Pubmed Central) - Apr 15, 2020
No abstract available "What PoNL do you feel" is a simple, quick and valid question, which correlates moderately with functional and disability scales in non-diabetic polyneuropathy, and might be incorporated in polyneuropathy assessment.
- | Journal: How Much Drool Is Too Much? (Pubmed Central) - Apr 15, 2020
There is a broad differential for a patient with respiratory failure, and careful physical examination and history are imperative to reduce morbidity and prevent mortality. After further evaluation and workup, the diagnosis is ultimately revealed.
- ||||| nipocalimab (M281) / J&J
Trial suspension: An Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients With Generalized Myasthenia Gravis (clinicaltrials.gov) - Apr 14, 2020
P2, N=60, Suspended, Sponsor: Momenta Pharmaceuticals, Inc.
After further evaluation and workup, the diagnosis is ultimately revealed. Recruiting --> Suspended
- | Bridion (sugammadex) / Merck (MSD)
Journal: Sugammadex and postoperative myasthenic crisis. (Pubmed Central) - Apr 14, 2020
Recruiting --> Suspended No abstract available
- || batoclimab (IMVT-1401) / Roivant
New P2 trial: A Study to Evaluate the Efficacy, Safety and PD and PK of HBM9161 in MG Patients (clinicaltrials.gov) - Apr 14, 2020
P2, N=30, Not yet recruiting, Sponsor: Harbour BioMed (Guangzhou) Co. Ltd.
- | Journal: A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. (Pubmed Central) - Apr 14, 2020
To the best of our knowledge, this is the first report of a dominant, heterozygous mutation in SCN4A causing a complex phenotype of congenital myopathy and myotonia with multiple congenital anomalies and unique muscle pathology findings. This case is another addition to the ever expanding phenotype of SCN4A mutations.
- || prednisone / Generic mfg.
Clinical, Journal: Response to Treatment in Pediatric Ocular Myasthenia Gravis. (Pubmed Central) - Apr 14, 2020
This case is another addition to the ever expanding phenotype of SCN4A mutations. Compared to GMG, OMG patients had earlier disease onset and improved outcomes after treatment.
- || Clinical, Retrospective data, Journal: Histotyping of Indian thymomas: A clinicopathologic study from north India. (Pubmed Central) - Apr 12, 2020
Resected thymic carcinomas were conspicuously absent in our study. More studies need to be done to establish the frequency and biology of TETs from India.
- | Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
Journal: Low dose rituximab every 6 months for the treatment of acetylcholine receptor positive refractory generalized myasthenia gravis. (Pubmed Central) - Apr 12, 2020
More studies need to be done to establish the frequency and biology of TETs from India. Low dose rituximab every 6 months is effective in treating refractory GMG patients.
- | Journal: Plasma exosomal miR-106a-5p expression in myasthenia gravis. (Pubmed Central) - Apr 12, 2020
Low dose rituximab every 6 months is effective in treating refractory GMG patients. Exosomal miR-106a-5p was expressed differently in different types of MG and was associated with MG severity.
- || Clinical, Journal: Myasthenic crisis demanding mechanical ventilation: A multicenter analysis of 250 cases. (Pubmed Central) - Apr 11, 2020
Exosomal miR-106a-5p was expressed differently in different types of MG and was associated with MG severity. No abstract available
- || Clinical, Journal: Prognostic predictors of remission in ocular myasthenia after thymectomy. (Pubmed Central) - Apr 11, 2020
However, only age at onset of 40 years or younger (HR: 4.117, 95% CI, 1.177-14.399, P=0.027) remained significant after multivariable analysis. CSR could be achieved in about 40% of OMG patients 5 years after thymectomy and is likely to be predicted by age at onset of 40 years or younger.
- | azathioprine orodispersible / Generic mfg.
Review, Journal: Myasthenia gravis and specific immunotherapy: monoclonal antibodies. (Pubmed Central) - Apr 10, 2020
In recent years, owing to an increasing understanding of MG pathogenesis the development and execution of clinical trials with novel biologics, including monoclonal antibodies (mAbs) that have demonstrated higher safety and more specificity, provide new opportunities for the treatment of MG. In this article, we review recent advances in MG pathogenesis and the mAbs that have been used for target-specific MG therapy.
- || methotrexate / Generic mfg.
Clinical, Journal: Methotrexate Polyglutamation in a Myasthenia Gravis Clinical Trial. (Pubmed Central) - Apr 9, 2020
There are strong correlations between MTXPG and the MG-ADL, QMG, and MGC. Long chain methotrexate polyglutamates correlate better with MG outcomes.
- | Journal: The MX-Helix of Muscle nAChR Subunits Regulates Receptor Assembly and Surface Trafficking. (Pubmed Central) - Apr 9, 2020
Together, these findings indicate that the subunit MX-helix contributes to receptor assembly and is required for normal expression of the AChR and function of the NMJ. In addition, specific determinants in the β and δ subunit MX-helix contribute to quality control of AChR expression by intracellular retention and ubiquitination of unassembled subunits, and by facilitating the appropriate glycosylation of assembled surface AChR.
- [VIRTUAL] AMPAR autoimmunity: neurological, oncological and serological accompaniments (Room Istanbul) - Apr 9, 2020 - Abstract #EAN2020EAN_2421;
Thymoma was the most common tumor. Accompanying neural autoantibodies are more frequent than previously reported and the autoantibody profile is a biomarker of the underlying neoplasia.
- mycophenolate mofetil / Generic mfg., azathioprine orodispersible / Generic mfg.
[VIRTUAL] The risk factors for developing refractory Myasthenia gravis (Room Budapest) - Apr 9, 2020 - Abstract #EAN2020EAN_2329;
Background and aims: Most patients with Myasthenia gravis (MG) are successfully treated with acetylcholinesterase inhibitors, corticosteroids, and/or steroid sparing agents such as azathioprine and mycophenolate mofetil. Female patients, early onset of the disease, and myasthenic crisis are the risk factors for developing refractory MG.
- pyridostigmine Bromide / Generic mfg.
[VIRTUAL] Myasthenia gravis in Poland – national healthcare database epidemiological study (Room Budapest) - Apr 9, 2020 - Abstract #EAN2020EAN_2328;
Our epidemiological data was drawn from the National Health Fund (NFZ) database; MG patient was defined as a person who received at least once medical service coded in ICD-10 as myasthenia gravis (G70) and at least 2 reimbursed prescriptions for pyridostigmine bromide (Mestinon ) or ambenonium chloride (Mytelase ) in 2 consecutive years... Our findings provide information on epidemiology of symptomatic Myasthenia Gravis in Poland and can serve as a tool to evaluate health care resources needed for MG patients.
- Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Safety and Effectiveness of Eculizumab for Patients with Generalized Myasthenia Gravis in Japan: Interim Analysis of Post- Marketing Surveillance (Room Budapest) - Apr 9, 2020 - Abstract #EAN2020EAN_2324;
Our findings provide information on epidemiology of symptomatic Myasthenia Gravis in Poland and can serve as a tool to evaluate health care resources needed for MG patients. In a real-world setting, eculizumab was effective and well tolerated for treatment of AChR+ gMG in adult Japanese patients who were refractory to IVIg or plasmapheresis.
- HBM9161 / Harbour BioMed
[VIRTUAL] A Phase 1 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of HBM9161 in Chinese healthy volunteers (Room: Screen A6) - Apr 9, 2020 - Abstract #EAN2020EAN_2116;
P1
HBM9161 is safe and well-tolerated at a dose up to 680mg in Chinese subjects. The data warrant further investigation for its effects in IgG-mediated autoimmune disorders including Myasthenia Gravis.
- [VIRTUAL] Serum immunoglobulin free-light chains in myasthenia gravis: a biomarker of B-cell activity? (Room: Screen A11) - Apr 9, 2020 - Abstract #EAN2020EAN_2074;
Kappa and lambda FLC resulted a sensitive marker of AchR-MG. Further investigations are need to evaluated their role as biomarkers of disease activity.
- [VIRTUAL] Myasthenia gravis associated with other autoimmune diseases - what we found out from our clinical practice? (Room: Screen A11) - Apr 9, 2020 - Abstract #EAN2020EAN_2069;
Further investigations are need to evaluated their role as biomarkers of disease activity. Screening for AD should be done in everyday practice at patients with an MG, because of the greater risk of developing another AD disease and it’s clinical implications.
- [VIRTUAL] 3 individuals with mutation in exon 1f of PLEC and myasthenic phenotype (Room: Screen A11) - Apr 9, 2020 - Abstract #EAN2020EAN_2067;
We further characterize LGMD R17 plectin- related phenotype in terms of myasthenic symptoms and muscle MRI. Our findings support the hypothesis of a crucial role of plectin 1f isoform in NMJ.
- [VIRTUAL] Anti-CASPR2 clinical phenotypes correlate with HLA and immunological features (Scientific Theatre) - Apr 9, 2020 - Abstract #EAN2020EAN_1725;
Our findings support the hypothesis of a crucial role of plectin 1f isoform in NMJ. Clinical, immunological, and genetic characteristics of CASPR2-Ab patients support the existence of 3 major disorders (LE, NMT, and MoS), suggesting distinct etiopathogeneses.
- Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] No change in risk of infection among NMOSD and refractory gMG patients treated with eculizumab: findings from two phase 3 studies and their extensions (Scientific Theatre) - Apr 9, 2020 - Abstract #EAN2020EAN_1723;
P3
Clinical, immunological, and genetic characteristics of CASPR2-Ab patients support the existence of 3 major disorders (LE, NMT, and MoS), suggesting distinct etiopathogeneses. In these complement-mediated neurological conditions, overall risk and types of infections were similar in the eculizumab and placebo groups, regardless of concomitant IST.
- [VIRTUAL] Efficacy and safety of non-steroidal immunosuppressive treatments in Generalized Myasthenia Gravis patients. A systematic review and meta-analysis (Room Paris) - Apr 9, 2020 - Abstract #EAN2020EAN_1683;
Treatment with tacrolimus, cyclosporine, cyclophosphamide, rituximab and eculizumab showed significant effects on the QMG score. Tacrolimus, rituximab and eculizumab stand out as the treatments with better efficacy and safety profile in generalized MG resistant to 1st-line treatments, although more studies with greater homogeneity are needed to draw conclusions that lead to algorithms of therapeutic decision.
- [VIRTUAL] AChR antibody positivity rate in ocular myasthenia gravis: a matter of age? (Room Paris) - Apr 9, 2020 - Abstract #EAN2020EAN_1678;
From our results, current AChR Ab sensitivity in OMG may be higher than generally thought. This finding was associated with a rising prevalence of late-onset cases, paralleling epidemiological changes in GMG.
- prednisone / Generic mfg.
[VIRTUAL] Rest or Exercise (RESTOREX) in Myasthenia Gravis: a randomized controlled trial (Room Paris) - Apr 9, 2020 - Abstract #EAN2020EAN_1677;
This finding was associated with a rising prevalence of late-onset cases, paralleling epidemiological changes in GMG. The study provides class II evidence of improved quality of life in mild to moderate MG by 30 min walk compared to rest
- efgartigimod IV (ARGX-113 IV) / argenx, Broteio
[VIRTUAL] Efgartigimod in Myasthenia Gravis: Phase 3 Trial Design (Room Paris) - Apr 9, 2020 - Abstract #EAN2020EAN_1676;
The study provides class II evidence of improved quality of life in mild to moderate MG by 30 min walk compared to rest Efficacy and safety findings will be reported at the conclusion of the trial.
- [VIRTUAL] Paraneoplastic and non paraneoplastic Lambert-Eaton Myasthenic Syndrome: a restrospective descriptive study (Room Paris) - Apr 9, 2020 - Abstract #EAN2020EAN_1675;
The electrophysiological pattern of PN LEMS seems to be more severe as non PN, for CAMP amplitude as well as decrement and increment. If confirmed in a validation cohort, the severity of the electrophysiological picture could be included in a prediction score of PN LEMS.
- azathioprine orodispersible / Generic mfg.
[VIRTUAL] Progressive multifocal leukoencephalopathy of the cerebellar peduncle in a patient with Myasthenia gravis immunosuppressed with azathioprine: Case report and review of the literature (Room: Screen B6) - Apr 9, 2020 - Abstract #EAN2020EAN_1468;
Immunosuppressants were discontinued, CSF JC-Virus copies dropped quickly and pembrolizumab was given, but he died 14 weeks after symptom onset. PML caused by azathioprine is rarely reported, though analysis from the US Adverse event reporting system calculated on reporting odds ratio of 15 for this drug.
- [VIRTUAL] The Clinical Efficacy of Intravenous Immunoglobulin in Neurology - A Retrospective Cohort Study at the Mater Misericordiae University Hospital (Room: Screen A6) - Apr 9, 2020 - Abstract #EAN2020EAN_1456;
IVIg use was supported for most indications when compared to international evidence-based guidelines. However IVIg was prescribed for several indications despite limited evidence of efficacy.
- methylprednisolone oral / Generic mfg.
[VIRTUAL] Progressive encephalomyelitis with rigidity and myoclonus with multiple autoantibodies as a first manifestation of thymoma (Room: Screen A6) - Apr 9, 2020 - Abstract #EAN2020EAN_1445;
A rapidly progressive stiff person syndrome in a young patient should be a red flag for PERM and it should not be tested only for GlyR antibodies. To conclude, PERM in the presence of multiple autoantibodies should always raise the suspicion pf thymoma.
- prednisone / Generic mfg.
[VIRTUAL] Myasthenia gravis in the elderly: evaluation of an Italian cohort. (Room: Screen A11) - Apr 9, 2020 - Abstract #EAN2020EAN_1397;
Among our population, there were no patients with MuSK related MG. MG type, comorbidities (hypertension, diabetes, glaucoma, osteoporosis) should be carefully considered for a positive outcome.
- [VIRTUAL] Determinants of quality of life in myasthenia gravis patients (Room: Screen A11) - Apr 9, 2020 - Abstract #EAN2020EAN_1394;
Our data support the need to address not only severity of MG but comorbidities, such as obesity or depression as well, to improve QoL. Family support, employment and moderate physical activity are important as well.
- [VIRTUAL] Results of plasmapheresis treatment due to neurological disease in a hospital- based series (Room: Screen B5) - Apr 9, 2020 - Abstract #EAN2020EAN_1210;
Although the adverse events were frequent, most are mild, with minimal intervention required. In patients without the desired initial response, plasmapheresis may be useful in achieving a better clinical outcome.
- [VIRTUAL] Dropped Head Syndrome: a clinical and electrophysiological study in six patients (Room: Screen A2) - Apr 9, 2020 - Abstract #EAN2020EAN_1187;
Accurate etiological diagnosis of DHS requires a thorough clinical and electrophysiological assessment and use of neostigmine test. Our series helped us advancing a diagnostic algorithm of DHC.
- [VIRTUAL] Role of next generation sequencing in the diagnosis of congenital neuromuscular diseases: the experience of an Italian centre (Room Helsinki) - Apr 9, 2020 - Abstract #EAN2020EAN_1056;
NGS panels reached a molecular diagnosis in a higher number of patients and identified overlapping genes and phenotypes; however, many patients remain without a molecular diagnosis: this will be fundamental for patient’s selection for treatments under development. Finally, we propose a diagnostic algorithm for congenital neuromuscular diseases.
- zilucoplan (RA101495) / UCB
[VIRTUAL] Zilucoplan, an investigational peptide inhibitor of complement component 5, blocked muscle weakness in a humanized passive transfer model of immune- mediated necrotizing myopathy (Room Helsinki) - Apr 9, 2020 - Abstract #EAN2020EAN_1054;
P2
In this study, we provide preclinical evidence of the protective role for C5 inhibition by Zilucoplan in a humanized murine model of IMNM... Our data establish a central role for C5 activation in a preclinical IMNM model, supporting the therapeutic evaluation of zilucoplan in an ongoing multicenter Phase 2 IMNM clinical trial (NCT04025632).
- [VIRTUAL] Clinical and serological characteristics of patients with double seronegative Myasthenia Gravis (HoC Room) - Apr 9, 2020 - Abstract #EAN2020EAN_1005;
Around 25% of SNMG patients had AChR or MuSK antibodies which were reliably detected with a combinatorial CBA, co-expressing AChR and MuSK. These data support the use of a combinatorial CBA for screening SNMG patient sera.
- [VIRTUAL] Clinical, pathological and prognostic heterogeneity in immune checkpoint inhibitors-induced myositis (HoC Room) - Apr 9, 2020 - Abstract #EAN2020EAN_1001;
Ocular muscles are frequently involved (4/4 in our series) regardless MG co-morbidity. Severity, course and prognosis are heterogeneous and apparently unrelated to different pathological patterns.
- [VIRTUAL] Immune-mediated neurotoxic syndromes related to immune checkpoint inhibitors: experience in a tertiary care center. (HoC Room) - Apr 9, 2020 - Abstract #EAN2020EAN_1000;
Of relevance, most of patients were early diagnosed and showed good outcomes after early treatment. Lethality was particularly notable among GMG patients.
- Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
[VIRTUAL] Efficacy and safety of Rituximab in myasthenia gravis: a multicentric real life study (Room Seville) - Apr 9, 2020 - Abstract #EAN2020EAN_978;
Our study corroborated the efficacy and safety of Rituximab. Some complementary studies are necessary to confirm the place of Rituximab in pharmacopeia of MG treatment and to establish the recommendations of infusion protocol.
- [VIRTUAL] The neglected IgG1-3 antibodies in MuSK myasthenia gravis: novel evidence for their pathogenicity (Room Seville) - Apr 9, 2020 - Abstract #EAN2020EAN_977;
They stimulate MuSK-DOK7 phosphorylation cascade but fail to induce fully-formed AChR clusters. These effects are likely due the result of divalent binding to MuSK compared with binding of monovalent IgG4.
- [VIRTUAL] Single-fiber electromyography as an integral part of the diagnostic work-up of myasthenia gravis (HoC Room) - Apr 9, 2020 - Abstract #EAN2020EAN_835;
These effects are likely due the result of divalent binding to MuSK compared with binding of monovalent IgG4. sSF-EMG demonstrated an increased diag- nostic benefit compared with RS in patients with seronegative MG.
- Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
[VIRTUAL] Refractory Morvan Syndrome Responding Dramatically to Rituximab (ePoster) - Apr 9, 2020 - Abstract #EAN2020EAN_690;
The diffuse fasciculations associated with severe pain required treatment with morphine, gabapentin and duloxetine and failed to respond to intravenous pulse steroid and to 2 courses of IVIG... Rituximab appears to be a very promising therapy for patients with anti-Caspr2 syndrome who failed to respond to steroids or IVIG.
- [VIRTUAL] Nocturnal sleep in myasthenia gravis (MG) patients (ePoster) - Apr 9, 2020 - Abstract #EAN2020EAN_670;
Statistically significant decrease in the total cycle time and sleep efficiency, reduction of the REM, increase of arousals was revealed in the main group. The results indicate fragmentation of sleep, violation of its structure, decrease in the quality of sleep in MG.