- |||||||||| [VIRTUAL] Expanding the phenotype of p.R1460W mutation in SCN4A gene: a family report (ePoster) - Apr 9, 2020 - Abstract #EAN2020EAN_660;
Our findings expand the phenotypical spectrum associated with the p.R1460W mutation in SCN4A, by reporting a novel phenotype and a novel inheritance pattern. Further investigation on related genes and epigenetic factors are mandatory in order to better define and understand the mechanisms underlying the variable clinical expression.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Trial completion date, Trial primary completion date: Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases (clinicaltrials.gov) - Apr 6, 2020 P2, N=40, Suspended, Conclusions These findings put more insights into understanding the interaction of pectolinarin and AchE, as well as Alzheimer's disease. Trial completion date: Dec 2021 --> Dec 2027 | Trial primary completion date: Mar 2020 --> Mar 2026
- |||||||||| Enrollment change, Trial completion date, Trial primary completion date: BETA-MG: Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis (clinicaltrials.gov) - Apr 5, 2020
P2/3, N=30, Recruiting, Trial completion date: Dec 2021 --> Dec 2027 | Trial primary completion date: Mar 2020 --> Mar 2026 N=50 --> 30 | Trial completion date: Apr 2020 --> Oct 2021 | Trial primary completion date: Apr 2020 --> Oct 2021
- |||||||||| fasudil / Generic mfg.
Journal: Inhibition of ROCK activity regulates the balance of Th1, Th17 and Treg cells in myasthenia gravis. (Pubmed Central) - Apr 4, 2020 Furthermore, Fasudil inhibited the proliferation of antigen-specific Th1 and Th17 cells, and inhibited CD4 + T cells differentiated into Th1 and Th17 through decreasing phosphorylated Stat1 and Stat3, but promoted Treg cell differentiation through increasing phosphorylated Stat5. We conclude that dysregulated ROCK activity may be involved in the pathogenic immune response of MG and inhibition of ROCK activity might serve as a novel treatment strategy for MG.
- |||||||||| Journal: Neurological diseases in pregnancy. (Pubmed Central) - Apr 1, 2020
With advances in research on therapeutic options, some women can achieve a safe pregnancy in the setting of chronic neurological disease. VIDEO ABSTRACT: http://links.lww.com/COOG/A45.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo, Rituxan Hycela (rituximab/hyaluronidase) / Biogen, Roche, Halozyme
Review, Journal: Immunosuppressive and Immunomodulatory Therapies for Neuromuscular Diseases Part II: New and Novel Agents. (Pubmed Central) - Mar 31, 2020 This second article focuses on new and novel immune-based therapies including: eculizumab, a complement inhibitor approved for acetylcholine receptor antibody-positive myasthenia gravis (MG); rituximab, a B-cell depletion therapy with evolving indications in neuromuscular diseases; and the subcutaneous formulation of immunoglobulin G (SCIG) that gained approval for use in chronic inflammatory demyelinating polyneuropathy (CIDP) in 2018. Finally, several novel antigen-specific drugs at different stages of investigation in neuromuscular disease are also reviewed.
- |||||||||| Trial completion date, Trial primary completion date: Spontaneous Coronary Artery Dissection (SCAD) and Autoimmunity (clinicaltrials.gov) - Mar 30, 2020
P=N/A, N=600, Recruiting, Our findings may provide further insight into the relationship between the genotype and phenotype in collagen-associated diseases and improve the clinical diagnosis of Prolyl endopeptidase-like deficiency. Trial completion date: Mar 2020 --> Mar 2021 | Trial primary completion date: Dec 2019 --> Dec 2020
- |||||||||| Journal: Seronegative myasthenic syndrome? (Pubmed Central) - Mar 29, 2020
Trial completion date: Mar 2020 --> Mar 2021 | Trial primary completion date: Dec 2019 --> Dec 2020 No abstract available
- |||||||||| Clinical, Journal: Putative Abscopal Effect in Three Patients Treated by Combined Radiotherapy and Modulated Electrohyperthermia. (Pubmed Central) - Mar 28, 2020
These preliminary observational findings lead to the generation of a hypothesis that this combination induces both an in-situ, tumor-specific immune reaction and an anti-self-autoimmune reaction, in at least a small proportion of patients, and of those who experience the auto-immune response, tumor response is a concomitant finding. Mechanisms underlying this phenomenon need to be investigated further.
- |||||||||| prednisolone / Generic mfg.
Clinical, Journal: Autoimmune hepatitis during preoperative chemotherapy in a patient with thymoma. (Pubmed Central) - Mar 20, 2020 Liver dysfunction was improved by steroid treatment using oral prednisolone, 30 mg daily, followed by tapering...This is a rare case of non-myasthenic thymoma complicated with AIH. AIH should be carefully considered in thymoma patients with liver dysfunction, since multidisciplinary treatment is required for invasive thymoma.
- |||||||||| Tirosint (levothyroxine) / IBSA Institute Biochemical SA, azathioprine orodispersible / Generic mfg.
A Case of Respiratory Failure with Recurrent Intubations in a Patient with Severe Hypothyroidism (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area E) - Mar 15, 2020 - Abstract #ATS2020ATS_10182; Patient was started on azathioprine, pyridostigmine, and steroids initially for myasthenia...Following initiation of levothyroxine therapy, ten days post-intubation, improvement in tongue and lip swelling were noted and patient was successfully extubated.DiscussionRespiratory failure in myxedema can be attributed to central or mechanical factors that is responsive to thyroxine replacement therapy...Symptoms of bulbar and respiratory muscle weakness, hypoventilation and hypoxia characteristic of myasthenia gravis can overlap with those of myxedema leading to difficulty ascertaining the etiology of respiratory failure. Prompt recognition of myxedema coma is crucial due to associated high mortality rates if treatment is not instituted.
- |||||||||| A Rare Case of Shared Paralysis (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area A) - Mar 15, 2020 - Abstract #ATS2020ATS_9593;
Treatment modalities for all forms of Flavivirus encephalitis are the same with ongoing research demonstrating some role of ribavirin, interferon alpha and IVIG. Prognosis is usually guarded which emphasizes more on the early recognition and timely initiation of appropriate treatment.
- |||||||||| Not in the Clear: A Case of Recurrent Thymoma (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area A) - Mar 15, 2020 - Abstract #ATS2020ATS_8764;
There are, however, cases of recurrent thymoma 10-15 years post-resection with the longest time to recurrence noted in the literature to be 32 years. While cases such as this patient’s are uncommon, recurrent thymoma should be kept within the differential of patients reporting respiratory or chest abnormalities despite being outside of the typical recurrence timeframe, particularly because earlier diagnosis of recurrence leads to improved outcomes.
- |||||||||| prednisone / Generic mfg., azathioprine orodispersible / Generic mfg.
Lambert Eaton Myasthenic Syndrome Presenting as Hypoventilation-Induced Right Heart Dysfunction (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area D) - Mar 15, 2020 - Abstract #ATS2020ATS_7135; Aggressive malignancy screening and treatment is critical to the management of these patients. Additional treatments include small molecules that enhance acetylcholine release and immune modulating drugs such as IVIG and azathioprine.
- |||||||||| carboplatin / Generic mfg., Keytruda (pembrolizumab) / Merck (MSD), pemetrexed / Generic mfg.
An Eye-Opening Complication: A Rare Case of Myasthenia Gravis Secondary to Immune Checkpoint Inhibitors (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area A) - Mar 15, 2020 - Abstract #ATS2020ATS_6422; The diagnosis of immune-related myasthenia gravis is challenging as many symptoms are subtle and can mimic more common medical conditions. Early recognition and treatment of immune checkpoint inhibitor-related myasthenia gravis is critical as this form of the disease is more aggressive than typical autoimmune myasthenia gravis with several fatal cases reported in the literature.
- |||||||||| prednisone / Generic mfg.
Spontaneous Super Mesenteric Artery Hemorrhage in a Patient with Myasthenia Gravis (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area G) - Mar 15, 2020 - Abstract #ATS2020ATS_5475; He was treated with IVIG for 6 days followed by high dose prednisone with improvement in his neurological exam...In addition, patient had additional risk factors present for spontaneous SMA rupture including advanced age, male sex and atherosclerosis4. In patients who receive treatment with IVIG, there should be close monitoring for adverse reactions, particularly coagulopathy.
- |||||||||| Keytruda (pembrolizumab) / Merck (MSD)
Necrotizing Lymphocytic Myocarditis and Neuromuscular Respiratory Failure Due to Immunotherapy with Pembrolizumab (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area G) - Mar 15, 2020 - Abstract #ATS2020ATS_5448; This case suggests possible concomitant drug-induced diaphragmatic necrosis and myocarditis, which have both previously been reported with Pembrolizumab. Regardless of whether the patient had drug-induced myasthenia gravis or diaphragmatic necrosis, this case highlights a rare and interesting cause of cardiogenic shock and respiratory failure.
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