- |||||||||| Review, Journal: Targeted therapies for congenital myasthenic syndromes: systematic review and steps towards a treatabolome. (Pubmed Central) - Apr 2, 2019
We perform a systematic review of the evidence for pharmacological treatment of each CMS type, gathering evidence from 207 studies of over 1000 patients and stratifying by genetic defect, as treatment varies depending on the underlying cause. We assess the strength and quality of the evidence and create a dataset that provides the foundation for a computer-aided system to enable clinicians to gain easier access to information about treatable variants and the evidence they need to consider.
- |||||||||| Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
Trial completion date, Trial primary completion date: Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG (clinicaltrials.gov) - Mar 29, 2019 P3, N=60, Recruiting, This study is ongoing. Trial completion date: May 2019 --> Sep 2019 | Trial primary completion date: Mar 2019 --> Aug 2019
- |||||||||| Retrospective data, Journal: Low Conversion Rate of Ocular to Generalized Myasthenia Gravis in Singapore. (Pubmed Central) - Mar 29, 2019
Trial completion date: May 2019 --> Sep 2019 | Trial primary completion date: Mar 2019 --> Aug 2019 The conversion rate of OMG to GMG in Asian patients is low, predicted by presence of AChR antibodies, presence of thymoma and positive RNS.
- |||||||||| Journal: Mechanical Ventilation (Pubmed Central) - Mar 28, 2019
I herein describe the utility of mechanical ventilation in patients with major pulmonary resection, myasthenia gravis, lung transplantation, and cardiac surgery, for each. We should understand not only the utility but also the non-physiological condition during mechanical ventilation.
- |||||||||| Clinical, Journal: Effect of Diaphragm Plication in Thymoma Patients with and without Myasthenia Gravis. (Pubmed Central) - Mar 28, 2019
These values were less in patients with myasthenia gravis and diaphragm plication than patients with myasthenia gravis and no diaphragm plication. Excision of the phrenic nerve in patients with myasthenia gravis associated with thymoma and phrenic nerve involvement is appropriate.
- |||||||||| Clinical, Journal: Pulmonary Function Testing in Elderly Patients Treated for a Myasthenia Gravis Exacerbation. (Pubmed Central) - Mar 28, 2019
In patients treated for a myasthenia gravis exacerbation, pulmonary function values are significantly reduced in elderly patients compared to nonelderly patients. Despite very low SVC and MIP values most elderly patients do not require intubation however they do require intensive monitoring for serious respiratory complications.
- |||||||||| mycophenolate sodium / generics
Clinical, Review, Journal: Mycophenolic acid in patients with immune-mediated inflammatory diseases: From humans to dogs. (Pubmed Central) - Mar 27, 2019 MMF became generically available in the United States in 2008, and its clinical utility is increasingly being explored as a treatment option for dogs with immune-mediated diseases. This review summarizes the available literature for MPA pharmacokinetics and pharmacodynamics, and the current status of MPA as a treatment for client-owned dogs diagnosed with immune-mediated diseases.
- |||||||||| Rituxan (rituximab) / Roche, Biogen
Journal: Myasthenic Crisis (Pubmed Central) - Mar 23, 2019 Hallmarks of therapy are symptomatic treatment with acetylcholine esterase inhibitors, plasmapheresis or immunoadsorption, polyvalent immunoglobulins, an early start of steroids and immunosuppressants, namely azathioprine, and a consequent escalation to monoclonal antibody therapy, with rituximab being the first choice. Special demands in ICU treatments are a structured weaning concept, consequent treatment of comorbidities and complications, first and foremost infections, dysphagia management, and comprehensive prevention and treatments of delirium.
- |||||||||| Review, Journal: Swallowing and Secretion Management in Neuromuscular Disease. (Pubmed Central) - Mar 18, 2019
Dysphagia may lead to medical complications, such as malnutrition, dehydration, aspiration pneumonia, and other pulmonary complications, as well as social isolation and reduced overall quality of life. This review provides an overview of dysphagia associated with neuromuscular disease in adults, along with a concise review of swallowing assessment and intervention options.
- |||||||||| Orencia (abatacept) / BMS
Trial completion date, Trial primary completion date: Trial of Orencia in Patients With Myasthenia Gravis (clinicaltrials.gov) - Feb 16, 2019 P1, N=20, Recruiting, This review provides an overview of dysphagia associated with neuromuscular disease in adults, along with a concise review of swallowing assessment and intervention options. Trial completion date: Jan 2019 --> Apr 2020 | Trial primary completion date: Jan 2019 --> Apr 2020
- |||||||||| Firdapse (amifampridine) / BioMarin, Catalyst Pharma
Trial completion date, Trial primary completion date: Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes (clinicaltrials.gov) - Dec 28, 2018 P3, N=20, Recruiting, Active, not recruiting --> Completed | Trial completion date: Nov 2019 --> Sep 2018 | Trial primary completion date: Sep 2019 --> Sep 2018 Trial completion date: Dec 2018 --> Dec 2019 | Trial primary completion date: Nov 2018 --> Jul 2019
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Trial primary completion date: Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases (clinicaltrials.gov) - Dec 20, 2018 P2, N=40, Recruiting, Trial completion date: Dec 2018 --> Dec 2019 | Trial primary completion date: Nov 2018 --> Jul 2019 Trial primary completion date: Dec 2018 --> Mar 2020
- |||||||||| Journal: The Integrated Genomic Landscape of Thymic Epithelial Tumors. (Pubmed Central) - Dec 14, 2018
We further observe enrichment of mutations in HRAS, NRAS, and TP53. Last, we identify a molecular link between thymoma and the autoimmune disease myasthenia gravis, characterized by tumoral overexpression of muscle autoantigens, and increased aneuploidy.
- |||||||||| Trial completion, Enrollment change: The Role of the Thymus in Myasthenia Gravis (clinicaltrials.gov) - Nov 29, 2018
P=N/A, N=20, Completed, Last, we identify a molecular link between thymoma and the autoimmune disease myasthenia gravis, characterized by tumoral overexpression of muscle autoantigens, and increased aneuploidy. Active, not recruiting --> Completed | N=80 --> 20
- |||||||||| bortezomib / Generic mfg.
Trial completion date, Trial primary completion date: Therapy of Antibody-mediated Autoimmune Diseases by Bortezomib (TAVAB) (clinicaltrials.gov) - Nov 29, 2018 P2, N=18, Recruiting, Active, not recruiting --> Completed | N=80 --> 20 Trial completion date: Dec 2018 --> Dec 2019 | Trial primary completion date: Dec 2018 --> Dec 2019
- |||||||||| Trial completion, Enrollment change, Trial completion date, Trial primary completion date: Myasthenia Gravis and Psyche (clinicaltrials.gov) - Nov 29, 2018
P=N/A, N=1399, Completed, Trial completion date: Dec 2018 --> Dec 2019 | Trial primary completion date: Dec 2018 --> Dec 2019 Recruiting --> Completed | N=4300 --> 1399 | Trial completion date: Dec 2018 --> Dec 2017 | Trial primary completion date: Dec 2018 --> Dec 2017
- |||||||||| NN1213 / Novo Nordisk
Biomarker, Enrollment status: Biomarkers in Neural Disorders (clinicaltrials.gov) - Oct 17, 2018 P=N/A, N=440, Enrolling by invitation, Recruiting --> Completed | N=4300 --> 1399 | Trial completion date: Dec 2018 --> Dec 2017 | Trial primary completion date: Dec 2018 --> Dec 2017 Recruiting --> Enrolling by invitation
|