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1. Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 Highly specialised technologies guidance Published: 27 November 2019 www.nice.org.uk/guidance/hst12 © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent the view of NICE, arrived at after careful (...) a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible. Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12) © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 2 of 32Contents Contents 1 Recommendations 4 2 The condition 6 3 The technology 7 4 Consideration of the evidence

2019 National Institute for Health and Clinical Excellence - Highly specialised technology

2. Intramuscular diaphragm stimulation for ventilator-dependent chronic respiratory failure caused by motor neurone disease

Intramuscular diaphragm stimulation for ventilator-dependent chronic respiratory failure caused by motor neurone disease Intr Intramuscular diaphr amuscular diaphragm stimulation for agm stimulation for v ventilator-dependent chronic respir entilator-dependent chronic respiratory failure atory failure caused b caused by motor neurone disease y motor neurone disease Interventional procedures guidance Published: 27 September 2017 nice.org.uk/guidance/ipg593 Y Y our responsibility our (...) of implementing NICE recommendations wherever possible. This guidance replaces IPG307. © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 1 of 71 1 Recommendations Recommendations 1.1 Current evidence on intramuscular diaphragm stimulation for ventilator- dependent chronic respiratory failure caused by motor neurone disease suggests that there are serious long-term safety concerns. Evidence on efficacy is limited and therefore

2017 National Institute for Health and Clinical Excellence - Interventional Procedures

3. Cerliponase alfa (Brineura) - the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency

Cerliponase alfa (Brineura) - the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency Search Page - Drug and Health Product Register Language selection Search and menus Search Search website Search Topics menu You are here: Summary Basis of Decision - - Health Canada Expand all Summary Basis of Decision (SBD) for Contact: Summary Basis of Decision (SBD) documents provide information related to the original authorization

2019 Health Canada - Drug and Health Product Register

4. Treatment of fatigue in amyotrophic lateral sclerosis/motor neuron disease. Full Text available with Trip Pro

Treatment of fatigue in amyotrophic lateral sclerosis/motor neuron disease. Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), is terminal, progressive neurological condition for which there are no curative treatments. Among people with ALS/MND, fatigue is a common and debilitating symptom, which is characterised by reversible motor weakness and whole-body tiredness that is only partially relieved by rest. The effectiveness of pharmacological or non-pharmacological

2018 Cochrane

5. Brineura - cerliponase alfa - Neuronal Ceroid Lipofuscinosis Type 2

Brineura - cerliponase alfa - Neuronal Ceroid Lipofuscinosis Type 2 cerliponase alfa | CADTH.ca Find the information you need cerliponase alfa cerliponase alfa Last Updated: June 28, 2019 Result type: Reports Project Number: SR0574-000 Product Line: Generic Name: cerliponase alfa Brand Name: Brineura Manufacturer: Biomarin Pharmaceutical (Canada) Inc. Indications: Neuronal Ceroid Lipofuscinosis Type 2 Manufacturer Requested Reimbursement Criteria 1 : Indicated for patients with CLN2 disease (...) , also known as tripeptidyl peptidase 1 (TPP1) deficiency or Neuronal Ceroid Lipofuscinosis type 2. Submission Type: New Project Status: Complete Biosimilar: No Companion Diagnostics: No Date Recommendation Issued: May 23, 2019 Recommendation Type: Reimburse with clinical criteria and/or conditions Fee Schedule: Schedule A The requested reimbursement criteria are provided by the applicant and do not necessarily reflect the views of CADTH. Reimbursement criteria from CADTH will be documented

2018 Canadian Agency for Drugs and Technologies in Health - Common Drug Review

7. Mapping neuronal inputs to Kiss1 neurons in the arcuate nucleus of the mouse. Full Text available with Trip Pro

Mapping neuronal inputs to Kiss1 neurons in the arcuate nucleus of the mouse. The normal function of the mammalian reproductive axis is strongly influenced by physiological, metabolic and environmental factors. Kisspeptin neuropeptides, encoded by the Kiss1 gene, are potent regulators of the mammalian reproductive axis by stimulating gonadodropin releasing hormone secretion from the hypothalamus. To understand how the reproductive axis is modulated by higher order neuronal inputs we have mapped (...) the afferent circuits into arcuate (ARC) Kiss1 neurons. We used a transgenic mouse that expresses the CRE recombinase in Kiss1 neurons for conditional viral tracing with genetically modified viruses. CRE-mediated activation of these viruses in Kiss1 neurons allows the virus to move transynaptically to label neurons with primary or secondary afferent inputs into the Kiss1 neurons. Several regions of the brain showed synaptic connectivity to arcuate Kiss1 neurons including proopiomelanocortin neurons

2019 PLoS ONE

8. Mechanical ventilation for amyotrophic lateral sclerosis/motor neuron disease. Full Text available with Trip Pro

Mechanical ventilation for amyotrophic lateral sclerosis/motor neuron disease. Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease, is a fatal neurodegenerative disease. Neuromuscular respiratory failure is the most common cause of death, which usually occurs within two to five years of the disease onset. Supporting respiratory function with mechanical ventilation may improve survival and quality of life. This is the second update of a review first published in 2009

2017 Cochrane

9. [Cerliponase alfa (neuronal ceroid lipofuscinosis) - assessment according to õ35a (para. 1., sentence 10) Social Code Book V]

[Cerliponase alfa (neuronal ceroid lipofuscinosis) - assessment according to õ35a (para. 1., sentence 10) Social Code Book V] Cerliponase alfa (neuronale zeroidlipofuszinose typ 2): bewertung gemäß § 35a Abs. 1 Satz 10 SGB V; dossierbewertung; auftrag G17-06 [Cerliponase alfa (neuronal ceroid lipofuscinosis) - assessment according to §35a (para. 1., sentence 10) Social Code Book V] Cerliponase alfa (neuronale zeroidlipofuszinose typ 2): bewertung gemäß § 35a Abs. 1 Satz 10 SGB V (...) ; dossierbewertung; auftrag G17-06 [Cerliponase alfa (neuronal ceroid lipofuscinosis) - assessment according to §35a (para. 1., sentence 10) Social Code Book V] Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen Record Status This is a bibliographic record of a published health technology assessment from a member of INAHTA. No evaluation of the quality of this assessment has been made for the HTA database. Citation Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen. Cerliponase alfa

2018 Health Technology Assessment (HTA) Database.

10. Symptomatic treatments for amyotrophic lateral sclerosis/motor neuron disease. Full Text available with Trip Pro

Symptomatic treatments for amyotrophic lateral sclerosis/motor neuron disease. Motor neuron disease (MND), which is also known as amyotrophic lateral sclerosis (ALS), causes a wide range of symptoms but the evidence base for the effectiveness of the symptomatic treatment therapies is limited.To summarise the evidence from Cochrane Systematic Reviews of all symptomatic treatments for MND.We searched the Cochrane Database of Systematic Reviews (CDSR) on 15 November 2016 for systematic reviews

2017 Cochrane

11. Uncovering Neuronal Networks Defined by Consistent Between-Neuron Spike Timing from Neuronal Spike Recordings Full Text available with Trip Pro

Uncovering Neuronal Networks Defined by Consistent Between-Neuron Spike Timing from Neuronal Spike Recordings It is widely assumed that distributed neuronal networks are fundamental to the functioning of the brain. Consistent spike timing between neurons is thought to be one of the key principles for the formation of these networks. This can involve synchronous spiking or spiking with time delays, forming spike sequences when the order of spiking is consistent. Finding networks defined (...) by their sequence of time-shifted spikes, denoted here as spike timing networks, is a tremendous challenge. As neurons can participate in multiple spike sequences at multiple between-spike time delays, the possible complexity of networks is prohibitively large. We present a novel approach that is capable of (1) extracting spike timing networks regardless of their sequence complexity, and (2) that describes their spiking sequences with high temporal precision. We achieve this by decomposing frequency-transformed

2018 eNeuro

12. Impact of insulin on primary arcuate neurons culture is dependent on early-postnatal nutritional status and neuronal subpopulation. Full Text available with Trip Pro

Impact of insulin on primary arcuate neurons culture is dependent on early-postnatal nutritional status and neuronal subpopulation. Nutrition plays a critical role in programming and shaping linear growth during early postnatal life through direct action on the development of the neuroendocrine somatotropic (GH/IGF-1) axis. IGF-1 is a key factor in modulating the programming of linear growth during this period. Notably, IGF-1 preferentially stimulates axonal growth of GHRH neurons (...) specific approach (GHRH-eGFP mice) under both physiological conditions (normally fed pups) and those of dietary restriction (underfed pups). Our data suggest that insulin failed to directly control axonal growth of Arc neurons or influence specific IGF-1-mediated effects on GHRH neurons. Insulin may act on neuronal welfare, which appears to be dependent on neuronal sub-populations and is influenced by the nutritional status of pups in which Arc neurons develop.

2018 PLoS ONE

13. Engrafted newborn neurons could functionally integrate into the host neuronal network Full Text available with Trip Pro

Engrafted newborn neurons could functionally integrate into the host neuronal network 28271666 2018 02 26 2018 11 13 2095-8137 38 1 2017 Jan 18 Zoological research Zool Res Engrafted newborn neurons could functionally integrate into the host neuronal network. 5-6 10.13918/j.issn.2095-8137.2017.005 Wang Zheng-Bo ZB Key Laboratory of Animal Models and Human Disease Mechanisms of Chinese Academy of Sciences & Yunnan Province, Kunming Institute of Zoology, Kunming Yunnan 650223, China. Qin Dong (...) Apr 24;157(3):726-39 24746791 Neuron. 2011 May 26;70(4):614-25 21609820 Cell Transplant. 2015;24(4):681-90 25839189 J Neurosci. 2004 Apr 28;24(17):4145-56 15115809 Nat Med. 1999 Dec;5(12):1410-2 10581084 Oxid Med Cell Longev. 2015;2015:618631 26146528 J Cereb Blood Flow Metab. 2016 Dec;36(12 ):2034-2043 27742890 Nature. 2002 Jul 4;418(6893):50-6 12077607 Nat Neurosci. 2013 Aug;16(8):1154-61 23792946 Int J Mol Sci. 2015 Nov 05;16(11):26473-92 26556344 Proc Natl Acad Sci U S A. 2011 Dec 13;108(50

2017 Zoological research

14. Cerliponase alfa (Brineura) - for treating neuronal ceroid lipofuscinosis type 2 (CLN2 disease)

Cerliponase alfa (Brineura) - for treating neuronal ceroid lipofuscinosis type 2 (CLN2 disease) 30 Churchill Place ? Canary Wharf ? London E14 5EU ? United Kingdom An agency of the European Union Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5520 Send a question via our website www.ema.europa.eu/contact 21 April 2017 EMA/31226/2017 Committee for Medicinal Products for Human Use (CHMP) Assessment report Brineura International non-proprietary name: cerliponase alfa Procedure No. EMEA/H/C (...) Isoelectric Focusing IT-C Intrathecal-cisternal IT-L Intrathecal-lumbar ITT Intent-to-treat IVC Intracerebroventricularly LCA Limit of in vitro cell age Assessment report EMA/312226/2017 Page 5/95 MALLs Multi angle laser light scattering MCB Master Cell Bank MedDRA Medical Dictionary for Regulatory Activities MRI Magnetic resonance imaging MRS Magnetic resonance spectroscopy NAA N-acetylaspartate Nab Neutralizing anti-TPP1 antibody NCI National Cancer Institute NCL Neuronal ceroid lipofuscinosis NOR

2017 European Medicines Agency - EPARs

15. Motor neurone disease: assessment and management

Motor neurone disease: assessment and management Motor neurone disease: assessment and Motor neurone disease: assessment and management management NICE guideline Published: 24 February 2016 nice.org.uk/guidance/ng42 © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guideline represent the view of NICE, arrived at after careful consideration (...) be inconsistent with complying with those duties. Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible. Motor neurone disease: assessment and management (NG42) © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 2 of 48Contents Contents Overview 4 Who

2016 National Institute for Health and Clinical Excellence - Clinical Guidelines

16. Expression-based decision tree model reveals distinct microRNA expression pattern in pediatric neuronal and mixed neuronal-glial tumors. Full Text available with Trip Pro

Expression-based decision tree model reveals distinct microRNA expression pattern in pediatric neuronal and mixed neuronal-glial tumors. The understanding of the molecular biology of pediatric neuronal and mixed neuronal-glial brain tumors is still insufficient due to low frequency and heterogeneity of those lesions which comprise several subtypes presenting neuronal and/or neuronal-glial differentiation. Important is that the most frequent ganglioglioma (GG) and dysembryoplastic

2019 BMC Cancer

17. USE of anti-neuronal antibodies to diagnose paraneoplastic neurological syndromes

USE of anti-neuronal antibodies to diagnose paraneoplastic neurological syndromes Use of anti-neuronal antibodies to diagnose paraneoplastic neurological syndromes Use of anti-neuronal antibodies to diagnose paraneoplastic neurological syndromes Gonzalez L, Augustovski F, Pichon-Riviere A, García Martí S, Alcaraz A, Bardach A, Ciapponi A, López A, Rey-Ares L Record Status This is a bibliographic record of a published health technology assessment from a member of INAHTA. No evaluation (...) of the quality of this assessment has been made for the HTA database. Citation Gonzalez L, Augustovski F, Pichon-Riviere A, García Martí S, Alcaraz A, Bardach A, Ciapponi A, López A, Rey-Ares L. Use of anti-neuronal antibodies to diagnose paraneoplastic neurological syndromes. Buenos Aires: Institute for Clinical Effectiveness and Health Policy (IECS). Informe de Respuesta Rapida No. 473. 2016 Authors' conclusions The evidence found supporting the use of anti-neuronal antibodies to diagnose paraneoplastic

2017 Health Technology Assessment (HTA) Database.

18. Platelets mediate protective neuroinflammation and promote neuronal plasticity at the site of neuronal injury. (Abstract)

Platelets mediate protective neuroinflammation and promote neuronal plasticity at the site of neuronal injury. It is generally accepted that inflammation within the CNS contributes to neurodegeneration after traumatic brain injury (TBI), but it is not clear how inflammation is initiated in the absence of infection and whether this neuroinflammation is predominantly beneficial or detrimental. We have previously found that brain-enriched glycosphingolipids within neuronal lipid rafts (NLR (...) ) induced platelet degranulation and secretion of neurotransmitters and pro-inflammatory factors. In the present study, we compared TBI-induced inflammation and neurodegeneration in wild-type vs. St3gal5 deficient (ST3-/-) mice that lack major CNS-specific glycosphingolipids. After TBI, microglial activation and CNS macrophage infiltration were substantially reduced in ST3-/- animals. However, ST3-/- mice had a larger area of CNS damage with marked neuronal/axonal loss. The interaction of platelets

2018 Brain, behavior, and immunity

19. Upregulation of neuronal astrocyte elevated gene-1 protects nigral dopaminergic neurons in vivo Full Text available with Trip Pro

Upregulation of neuronal astrocyte elevated gene-1 protects nigral dopaminergic neurons in vivo The role of astrocyte elevated gene-1 (AEG-1) in nigral dopaminergic (DA) neurons has not been studied. Here we report that the expression of AEG-1 was significantly lower in DA neurons in the postmortem substantia nigra of patients with Parkinson's disease (PD) compared to age-matched controls. Similarly, decreased AEG-1 levels were found in the 6-hydroxydopamine (6-OHDA) mouse model of PD. An adeno (...) -associated virus-induced increase in the expression of AEG-1 attenuated the 6-OHDA-triggered apoptotic death of nigral DA neurons. Moreover, the neuroprotection conferred by the AEG-1 upregulation significantly intensified the neurorestorative effects of the constitutively active ras homolog enriched in the brain [Rheb(S16H)]. Collectively, these results demonstrated that the sustained level of AEG-1 as an important anti-apoptotic factor in nigral DA neurons might potentiate the therapeutic effects

2018 Cell death & disease

20. Automated sorting of neuronal trees in fluorescent images of neuronal networks using NeuroTreeTracer Full Text available with Trip Pro

Automated sorting of neuronal trees in fluorescent images of neuronal networks using NeuroTreeTracer Fluorescence confocal microscopy has become increasingly more important in neuroscience due to its applications in image-based screening and profiling of neurons. Multispectral confocal imaging is useful to simultaneously probe for distribution of multiple analytes over networks of neurons. However, current automated image analysis algorithms are not designed to extract single-neuron arbors (...) in images where neurons are not separated, hampering the ability map fluorescence signals at the single cell level. To overcome this limitation, we introduce NeuroTreeTracer - a novel image processing framework aimed at automatically extracting and sorting single-neuron traces in fluorescent images of multicellular neuronal networks. This method applies directional multiscale filters for automated segmentation of neurons and soma detection, and includes a novel tracing routine that sorts neuronal trees

2018 Scientific reports

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