24 research outputs found

    Guideline for the management of myasthenic syndromes

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    Myasthenia gravis (MG), Lambert-Eaton myasthenic syndrome (LEMS), and congenital myasthenic syndromes (CMS) represent an etiologically heterogeneous group of (very) rare chronic diseases. MG and LEMS have an autoimmune-mediated etiology, while CMS are genetic disorders. A (strain dependent) muscle weakness due to neuromuscular transmission disorder is a common feature. Generalized MG requires increasingly differentiated therapeutic strategies that consider the enormous therapeutic developments of recent years. To include the newest therapy recommendations, a comprehensive update of the available German-language guideline ‘Diagnostics and therapy of myasthenic syndromes’ has been published by the German Neurological society with the aid of an interdisciplinary expert panel. This paper is an adapted translation of the updated and partly newly developed treatment guideline. It defines the rapid achievement of complete disease control in myasthenic patients as a central treatment goal. The use of standard therapies, as well as modern immunotherapeutics, is subject to a staged regimen that takes into account autoantibody status and disease activity. With the advent of modern, fast-acting immunomodulators, disease activity assessment has become pivotal and requires evaluation of the clinical course, including severity and required therapies. Applying MG-specific scores and classifications such as Myasthenia Gravis Activities of Daily Living, Quantitative Myasthenia Gravis, and Myasthenia Gravis Foundation of America allows differentiation between mild/moderate and (highly) active (including refractory) disease. Therapy decisions must consider age, thymic pathology, antibody status, and disease activity. Glucocorticosteroids and the classical immunosuppressants (primarily azathioprine) are the basic immunotherapeutics to treat mild/moderate to (highly) active generalized MG/young MG and ocular MG. Thymectomy is indicated as a treatment for thymoma-associated MG and generalized MG with acetylcholine receptor antibody (AChR-Ab)-positive status. In (highly) active generalized MG, complement inhibitors (currently eculizumab and ravulizumab) or neonatal Fc receptor modulators (currently efgartigimod) are recommended for AChR-Ab-positive status and rituximab for muscle-specific receptor tyrosine kinase (MuSK)-Ab-positive status. Specific treatment for myasthenic crises requires plasmapheresis, immunoadsorption, or IVIG. Specific aspects of ocular, juvenile, and congenital myasthenia are highlighted. The guideline will be further developed based on new study results for other immunomodulators and biomarkers that aid the accurate measurement of disease activity

    Scientific Assessment for Urban Air Mobility (UAM)

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    Better connecting the international research community and the International Civil Aviation Organization (ICAO) enables effective assessments of novel aviation innovations. The International Forum for Aviation Research (IFAR) created a group on Urban Air Mobility (UAM) to explore the broad array of aspects relevant to the ICAO mandate. The assessment began with a study of the current industry landscape, including an overview of existing market studies, proposed aircraft designs and concepts, and potential paths of industry evolution. The Industry Assessment is summarized into key takeaways highlighting the need for international assessments on economic and societal factors associated with UAM, common understanding of the extent to which the nascent industry can leverage current infrastructure and regulatory structures, and harmonization of industrywide terminology. The subsequent Scientific Assessment, developed through cooperative efforts between international domain experts, captures 17 focus areas relevant to UAM. All focus areas present opportunities for further research. Key takeaways include: the need for further study of the impact of autonomous systems (AS) on the industry; infrastructure requirements (including vertiports and weather sensing) to support the industry; and data requirements (including domains such as cybersecurity, emissions, and safety) to ensure safe, scalable operations. Finally, a brief overview of the current standards landscape as relevant to the Scientific Assessment is presented, which displays the benefits of applying digital systems engineering techniques to map current research efforts to ongoing standards activities

    Rechtliche Situation von Off-Label Use bis Heilversuch

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    Study protocol: the effect of whole body vibration on acute unilateral unstable lateral ankle sprain- a biphasic randomized controlled trial

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    Abstract Background Ankle sprains often result in ankle instability, which is most likely caused by damage to passive structures and neuromuscular impairment. Whole body vibration (WBV) is a neuromuscular training method improving those impaired neurologic parameters. The aim of this study is to compare the current gold standard functional treatment to functional treatment plus WBV in patients with acute unilateral unstable inversion ankle sprains. Methods/Design 60 patients, aged 18–40 years, presenting with an isolated, unilateral, acute unstable inversion ankle sprain will be included in this bicentric, biphasic, randomized controlled trial. Samples will be randomized by envelope drawing. All patients will be allowed early mobilization and pain-dependent weight bearing, limited functional immobilization by orthosis, PRICE, NSARDs as well as home and supervised physiotherapy. Supervised physical therapy will take place twice a week, for 30 minutes for a period of 6 weeks, following a standardized intervention protocol. During supervised physical therapy, the intervention group will perform exercises similar to those of the control group, on a side-alternating sinusoidal vibration platform. Two time-dependent primary outcome parameters will be assessed: short-term outcome after six weeks will be postural control quantified by the sway index; mid-term outcome after one year will be assessed by subjective instability, defined by the presence of giving-way attacks. Secondary outcome parameters include: return to pre-injury level of activities, residual pain, recurrence, objective instability, energy/coordination, Foot and Ankle Disability Index and EQ 5D. Discussion This is the first trial investigating the effects of WBV in patients with acute soft tissue injury. Inversion ankle sprains often result in ankle instability, which is most likely due to damage of neurological structures. Due to its unique, frequency dependent, influence on various neuromuscular parameters, WBV is a promising treatment method for patients with acute unstable inversion ankle sprains. Trial registration NCT01702597</p

    KNIME: The Konstanz Information Miner

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    The Konstanz Information Miner is a modular environment, which enables easy visual assembly and interactive execution of a data pipeline. It is designed as a teaching, research and collaboration platform, which enables simple integration of new algorithms and tools as well as data manipulation or visualization methods in the form of new modules or nodes. In this paper we describe some of the design aspects of the underlying architecture and briefly sketch how new nodes can be incorporated

    Open Source Data Pipelining für Interaktive Datenexploration

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    Der Konstanz Information Miner - KNIME - ist eine modulare Daten-Analyse Umgebung, die ein einfaches interaktives Erstellen und Ausführen datenflußorientierter Pipelines erlaubt. KNIME bietet als Lern-, Forschungs- und Kollaborations-Software eine ideale Plattform zur Anwendung von Daten-, Transformations-, Visualisierungs- und Data-Mining Knoten. Durch seine erweiterbaren Schnittstellen ist es leicht möglich, neue Algorithmen, aber auch bestehende Tools zu integrieren - u.a. sind Weka, das R-Project und CDK (Chemistry Development Kit) in KNIME verfügbar
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