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Relevant bibliographies by topics / TREAT-NMD

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Academic literature on the topic 'TREAT-NMD'

Author: Grafiati

Published: 4 June 2025

Last updated: 14 July 2025

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Journal articles on the topic "TREAT-NMD"

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Nguyen, Tran M., Matt Downs, Neil Bennett, et al. "Academic Productivity from Rare Neuromuscular Disease Registries: A Systematic Review." Journal of Rare Diseases Research & Treatment 7, no. 2 (2022): 5–15. http://dx.doi.org/10.29245/2572-9411/2022/2.1204.

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Background: TREAT-NMD is a global neuromuscular (NM) organization, created to enhance infrastructure to facilitate novel therapeutics reaching patients. One main activity is aimed at supporting NM disease registries. These rare disease registries are useful to fill knowledge gaps for various stakeholders in the disease community using real world data. Although it is important to understand how patient data is being utilized in the TREAT-NMD network and other rare disease registries, there is no systematic process or consistent metric for documenting the academic output from these registries. O

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Leary, Rebecca, Anne Oyewole, Katharine Bushby, and Annemieke Aartsma-Rus. "Translational Research in Europe for the Assessment and Treatment for Neuromuscular Disorders (TREAT-NMD)." Neuropediatrics 48, no. 04 (2017): 211–20. http://dx.doi.org/10.1055/s-0037-1604110.

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AbstractTranslational research in Europe for the assessment and treatment of neuromuscular disorders (TREAT-NMD) is a global network of world-class experts within the neuromuscular community whose mission is to support all stages of therapy development and improve the health and quality of life of people around the world with neuromuscular disorders (NMD). Since 2007, TREAT-NMD has played a central role in bringing together the right experts, patients, advocacy organizations, scientists, healthcare professionals, and pharmaceutical companies. By uniting these experts within the neuromuscular c

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Echols, Josh, Amna Siddiqui, Yanying Dai, et al. "A regulated NMD mouse model supports NMD inhibition as a viable therapeutic option to treat genetic diseases." Disease Models & Mechanisms 13, no. 8 (2020): dmm044891. http://dx.doi.org/10.1242/dmm.044891.

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ABSTRACTNonsense-mediated mRNA decay (NMD) targets mRNAs that contain a premature termination codon (PTC) for degradation, preventing their translation. By altering the expression of PTC-containing mRNAs, NMD modulates the inheritance pattern and severity of genetic diseases. NMD also limits the efficiency of suppressing translation termination at PTCs, an emerging therapeutic approach to treat genetic diseases caused by in-frame PTCs (nonsense mutations). Inhibiting NMD may help rescue partial levels of protein expression. However, it is unclear whether long-term, global NMD attenuation is sa

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Ambrosini, Anna, Daniela Calabrese, Francesco Maria Avato, et al. "The Italian neuromuscular registry: a coordinated platform where patient organizations and clinicians collaborate for data collection and multiple usage." Orphanet Journal of Rare Diseases 13, no. 1 (2018): 176. https://doi.org/10.1186/s13023-018-0918-z.

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<strong>Background: </strong>The worldwide landscape of patient registries in the neuromuscular disease (NMD) field has significantly changed in the last 10 years, with the international TREAT-NMD network acting as strong driver. At the same time, the European Medicines Agency and the large federations of rare disease patient organizations (POs), such as EURORDIS, contributed to a great cultural change, by promoting a paradigm shift from product-registries to patient-centred registries. In Italy, several NMD POs and Fondazione Telethon undertook the development of a TREAT-NMD linked patient re

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Wu, Xingxin, Tao Tan, and Qiang Xu. "Metastatic colorectal cancer cells harness nonsense-mediated mRNA decay for immune evasion." Journal of Immunology 204, no. 1_Supplement (2020): 242.17. http://dx.doi.org/10.4049/jimmunol.204.supp.242.17.

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Abstract Immunosuppression microenvironment allows primary tumor growth, while tumor-inherent factors that bring about immune evasion during metastasis remain elusive. Here, we observed a stronger nonsense-mediated mRNA decay (NMD) activity with a higher expression of up-frameshift protein 1 (UPF1) in colorectal cancer (CRC) metastasis than in matched primary cancer cells. In metastatic CRC SW620 cells, the higher UPF1 expression was found to arise from the increased stability of UPF1 by ubiquitin specific peptidase 10 (USP10)-mediated deubiquitination. In contrast, in primary CRC SW480 cells,

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Stanescu, A., J. Kirschner, C. Marx, et al. "T.P.3.08 The TREAT-NMD Clinical Trials Coordination Centre (CTCC)." Neuromuscular Disorders 18, no. 9-10 (2008): 796. http://dx.doi.org/10.1016/j.nmd.2008.06.247.

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Alkufi, Hussein Kadhum, and Hanan Jalal Kassab. "Nanospanlastic in situ Gel for Nose to Brain Delivery of Nimodipine: In vitro Optimization and in vivo Pharmacokinetic Study." Al-Rafidain Journal of Medical Sciences ( ISSN 2789-3219 ) 8, no. 1 (2025): 97–105. https://doi.org/10.54133/ajms.v8i1.1687.

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Background: The FDA has approved the medication nimodipine (NMD) to treat vasospasm brought on by subarachnoid hemorrhage. The most popular way to administer NMD is intravenously, which can result in several adverse effects, including bradycardia, hypotension, arrhythmias, and inflammation at the administration site. Objective: To evaluate the effectiveness of nose-to-brain (NTB) delivery of NMD as spanlastic nanovesicles (SNV) in situ gel into the brain and compare it with IV infusion. Methods: The nanovesicle formulation by the ethanol injection method used Span 60 as a non-ionic surfactant

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Gramsch, K., A. Pohl, J. Kirschner, R. Korinthenberg, S. Geismann, and A. Tassoni. "M.P.1.09 TREAT-NMD Clinical Trials Coordination Centre: Efficiency of networking." Neuromuscular Disorders 19, no. 8-9 (2009): 548–49. http://dx.doi.org/10.1016/j.nmd.2009.06.020.

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9

Hussein K. Alkufi and Hanan Kassab. "A Potential Method for Enhanced Performance of Nimodipine by Spanlastic Nanovesicle with Tween 40 as Edge Activator." Iraqi Journal of Pharmaceutical Sciences 34, no. 2 (2025): 227–38. https://doi.org/10.31351/vol34iss2pp227-238.

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This investigation aimed to investigate the potential of utilizing nanospanlastics to improve the bioavailability of nimodipine (NMD). To treat subarachnoid hemorrhage-induced vasospasm, physicians prescribe NMD, an FDA-approved drug. Intravenous (IV) administration is the most common way to provide NM, and it can have several unfavorable effects, including injection site irritation, bradycardia, hypotension, and arrhythmias. Made up of surfactants and edge activators (EAs), nanospanlastics are malleable nanovesicles. EAs improve the deformability of spanlastics by acting as a stabilizing forc

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Aartsma-Rus, A., E. Hoffman, F. Bucella, et al. "TREAT-NMD (translational research in Europe, assessment and treatment for neuromuscular disorders)." Neuromuscular Disorders 25 (October 2015): S271. http://dx.doi.org/10.1016/j.nmd.2015.06.309.

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Book chapters on the topic "TREAT-NMD"

1

Sejerson, Thomas, and Kate Bushby. "Standards of Care for Duchenne Muscular Dystrophy: Brief Treat-NMD Recommendations." In Advances in Experimental Medicine and Biology. Springer Netherlands, 2009. http://dx.doi.org/10.1007/978-90-481-2813-6_2.

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Reports on the topic "TREAT-NMD"

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Kastreva, Kristina, and Ivailo Tournev. Clinical Data Analysis of the Bulgarian Patient Registry for Myotonic Dystrophy Type 1 and Type 2 – Part of the Global TREAT-NMD Registry. "Prof. Marin Drinov" Publishing House of Bulgarian Academy of Sciences, 2020. http://dx.doi.org/10.7546/crabs.2020.06.18.

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