keyword
MENU ▼
Read by QxMD icon Read
search

clinical trials ALS

keyword
https://www.readbyqxmd.com/read/27896976/missing-data-imputation-in-the-electronic-health-record-using-deeply-learned-autoencoders
#1
Brett K Beaulieu-Jones, Jason H Moore
Electronic health records (EHRs) have become a vital source of patient outcome data but the widespread prevalence of missing data presents a major challenge. Different causes of missing data in the EHR data may introduce unintentional bias. Here, we compare the effectiveness of popular multiple imputation strategies with a deeply learned autoencoder using the Pooled Resource Open-Access ALS Clinical Trials Database (PRO-ACT). To evaluate performance, we examined imputation accuracy for known values simulated to be either missing completely at random or missing not at random...
2016: Pacific Symposium on Biocomputing
https://www.readbyqxmd.com/read/27844032/predicting-disease-progression-in-amyotrophic-lateral-sclerosis
#2
Albert A Taylor, Christina Fournier, Meraida Polak, Liuxia Wang, Neta Zach, Mike Keymer, Jonathan D Glass, David L Ennist
OBJECTIVE: It is essential to develop predictive algorithms for Amyotrophic Lateral Sclerosis (ALS) disease progression to allow for efficient clinical trials and patient care. The best existing predictive models rely on several months of baseline data and have only been validated in clinical trial research datasets. We asked whether a model developed using clinical research patient data could be applied to the broader ALS population typically seen at a tertiary care ALS clinic. METHODS: Based on the PRO-ACT ALS database, we developed random forest (RF), pre-slope, and generalized linear (GLM) models to test whether accurate, unbiased models could be created using only baseline data...
November 2016: Annals of Clinical and Translational Neurology
https://www.readbyqxmd.com/read/27830492/transcranial-magnetic-stimulation-for-the-assessment-of-neurodegenerative-disease
#3
REVIEW
Steve Vucic, Matthew C Kiernan
Transcranial magnetic stimulation (TMS) is a noninvasive technique that has provided important information about cortical function across an array of neurodegenerative disorders, including Alzheimer's disease, frontotemporal dementia, Parkinson's disease, and related extrapyramidal disorders. Application of TMS techniques in neurodegenerative diseases has provided important pathophysiological insights, leading to the development of pathogenic and diagnostic biomarkers that could be used in the clinical setting and therapeutic trials...
November 9, 2016: Neurotherapeutics: the Journal of the American Society for Experimental NeuroTherapeutics
https://www.readbyqxmd.com/read/27822919/cell-based-therapies-for-amyotrophic-lateral-sclerosis-motor-neuron-disease
#4
REVIEW
S Fadilah Abdul Wahid, Zhe Kang Law, Nor Azimah Ismail, Raymond Azman Ali, Nai Ming Lai
BACKGROUND: Amyotrophic lateral sclerosis (ALS), which is also known as motor neuron disease (MND) is a fatal disease associated with rapidly progressive disability, for which no definitive treatment as yet exists. Current treatment regimens largely focus on relieving symptoms to improve the quality of life of those affected. Based on data from preclinical studies, cell-based therapy is a promising treatment for ALS/MND. OBJECTIVES: To assess the effects of cell-based therapy for people with ALS/MND, compared with placebo or no additional treatment...
November 8, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27784774/safety-of-intrathecal-autologous-adipose-derived-mesenchymal-stromal-cells-in-patients-with-als
#5
Nathan P Staff, Nicolas N Madigan, Jonathan Morris, Mark Jentoft, Eric J Sorenson, Greg Butler, Dennis Gastineau, Allan Dietz, Anthony J Windebank
OBJECTIVE: To determine the safety of intrathecal autologous adipose-derived mesenchymal stromal cell treatment for amyotrophic lateral sclerosis (ALS). METHODS: Participants with ALS were enrolled and treated in this phase I dose-escalation safety trial, ranging from 1 × 10(7) (single dose) to 1 × 10(8) cells (2 monthly doses). After intrathecal treatments, participants underwent standardized follow-up, which included clinical examinations, revised ALS Functional Rating Scale (ALSFRS-R) questionnaire, blood and CSF sampling, and MRI of the neuroaxis...
November 22, 2016: Neurology
https://www.readbyqxmd.com/read/27778156/factors-predicting-survival-in-als-a-multicenter-italian-study
#6
Andrea Calvo, Cristina Moglia, Christian Lunetta, Kalliopi Marinou, Nicola Ticozzi, Gianluca Drago Ferrante, Carlo Scialo, Gianni Sorarù, Francesca Trojsi, Amelia Conte, Yuri M Falzone, Rosanna Tortelli, Massimo Russo, Adriano Chiò, Valeria Ada Sansone, Gabriele Mora, Vincenzo Silani, Paolo Volanti, Claudia Caponnetto, Giorgia Querin, Maria Rosaria Monsurrò, Mario Sabatelli, Nilo Riva, Giancarlo Logroscino, Sonia Messina, Nicola Fini, Jessica Mandrioli
The aim of this multicenter, retrospective study is to investigate the role of clinical characteristics and therapeutic intervention on ALS prognosis. The study included patients diagnosed from January 1, 2009 to December 31, 2013 in 13 Italian referral centers for ALS located in 10 Italian regions. Caring neurologists collected a detailed phenotypic profile and follow-up data until death into an electronic database. One center collected also data from a population-based registry for ALS. 2648 incident cases were collected...
October 24, 2016: Journal of Neurology
https://www.readbyqxmd.com/read/27752938/neuroimaging-endpoints-in-amyotrophic-lateral-sclerosis
#7
Ricarda A L Menke, Federica Agosta, Julian Grosskreutz, Massimo Filippi, Martin R Turner
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative, clinically heterogeneous syndrome pathologically overlapping with frontotemporal dementia. To date, therapeutic trials in animal models have not been able to predict treatment response in humans, and the revised ALS Functional Rating Scale, which is based on coarse disability measures, remains the gold-standard measure of disease progression. Advances in neuroimaging have enabled mapping of functional, structural, and molecular aspects of ALS pathology, and these objective measures may be uniquely sensitive to the detection of propagation of pathology in vivo...
October 17, 2016: Neurotherapeutics: the Journal of the American Society for Experimental NeuroTherapeutics
https://www.readbyqxmd.com/read/27751623/phrenic-nerve-stimulation-is-more-sensitive-than-ultrasound-measurement-of-diaphragm-thickness-in-assessing-early-als-progression
#8
Susana Pinto, Pedro Alves, Michael Swash, Mamede de Carvalho
AIMS OF THE STUDY: To compare the assessment of respiratory decline with conventional tests, measurement of diaphragm compound muscle action potential (CMAP) to phrenic nerve stimulation and diaphragm thickness to ultrasound (US) investigation in amyotrophic lateral sclerosis (ALS) patients followed for a short period of time. PATIENTS AND METHODS: We evaluated in 40 consecutive ALS patients, the clinical functional scale (ALSFRS-R), forced vital capacity (FVC), maximal voluntary ventilation (MVV), maximal inspiratory (MIP) and expiratory (MEP) pressures, sniff nasal inspiratory pressure (SNIP), Diaphragm-CMAP (latency and amplitude) and diaphragm US (maximal and minimal diaphragm thickness during full inspiration and expiration, respectively)...
October 14, 2016: Neurophysiologie Clinique, Clinical Neurophysiology
https://www.readbyqxmd.com/read/27751553/early-diaphragm-pacing-in-patients-with-amyotrophic-lateral-sclerosis-respistimals-a-randomised-controlled-triple-blind-trial
#9
Jésus Gonzalez-Bermejo, Capucine Morélot-Panzini, Marie-Laure Tanguy, Vincent Meininger, Pierre-François Pradat, Timothée Lenglet, Gaëlle Bruneteau, Nadine Le Forestier, Philippe Couratier, Nathalie Guy, Claude Desnuelle, Hélène Prigent, Christophe Perrin, Valérie Attali, Catherine Fargeot, Marie-Cécile Nierat, Catherine Royer, Fabrice Ménégaux, François Salachas, Thomas Similowski
BACKGROUND: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder associated with respiratory muscle weakness and respiratory failure. Non-invasive ventilation alleviates respiratory symptoms and prolongs life, but is a palliative intervention. Slowing the deterioration of diaphragm function before respiratory failure would be desirable. We aimed to assess whether early diaphragm pacing could slow down diaphragm deterioration and would therefore delay the need for non-invasive ventilation...
November 2016: Lancet Neurology
https://www.readbyqxmd.com/read/27744022/semi-supervised-learning-of-the-electronic-health-record-for-phenotype-stratification
#10
Brett K Beaulieu-Jones, Casey S Greene
Patient interactions with health care providers result in entries to electronic health records (EHRs). EHRs were built for clinical and billing purposes but contain many data points about an individual. Mining these records provides opportunities to extract electronic phenotypes, which can be paired with genetic data to identify genes underlying common human diseases. This task remains challenging: high quality phenotyping is costly and requires physician review; many fields in the records are sparsely filled; and our definitions of diseases are continuing to improve over time...
October 12, 2016: Journal of Biomedical Informatics
https://www.readbyqxmd.com/read/27720637/intraspinal-stem-cell-transplantation-for-amyotrophic-lateral-sclerosis-ready-for-efficacy-clinical-trials
#11
Nazem Atassi, Ettore Beghi, Miguel Blanquer, Nicholas M Boulis, Roberto Cantello, Claudia Caponnetto, Adriano Chiò, Stephen B Dunnett, Eva L Feldman, Angelo Vescovi, Letizia Mazzini
Intraspinal stem cell (SC) transplantation represents a new therapeutic approach for amyotrophic lateral sclerosis (ALS) clinical trials. There are considerable difficulties in designing future efficacy trials, some related to the field of ALS and some that are specific to SCs or the mode of delivery. In October 2015, the most controversial points on SC transplantation were addressed during an international workshop intended to bring together international SC and ALS researchers in a public discussion on a topic for which expertise is limited...
October 6, 2016: Cytotherapy
https://www.readbyqxmd.com/read/27716798/microneurotrophins-improve-survival-in-motor-neuron-astrocyte-co-cultures-but-do-not-improve-disease-phenotypes-in-a-mutant-sod1-mouse-model-of-amyotrophic-lateral-sclerosis
#12
Kelly E Glajch, Laura Ferraiuolo, Kaly A Mueller, Matthew J Stopford, Varsha Prabhkar, Achille Gravanis, Pamela J Shaw, Ghazaleh Sadri-Vakili
Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease caused by loss of motor neurons. ALS patients experience rapid deterioration in muscle function with an average lifespan of 3-5 years after diagnosis. Currently, the most effective therapeutic only extends lifespan by a few months, thus highlighting the need for new and improved therapies. Neurotrophic factors (NTFs) are important for neuronal development, maintenance, and survival. NTF treatment has previously shown efficacy in pre-clinical ALS models...
2016: PloS One
https://www.readbyqxmd.com/read/27677562/serum-urate-at-trial-entry-and-als-progression-in-empower
#13
ÉIlis J O'Reilly, Dawei Liu, Donald R Johns, Merit E Cudkowicz, Sabrina Paganoni, Michael A Schwarzschild, Melanie Leitner, Alberto Ascherio
Our objective was to determine whether serum urate predicts ALS progression. A study population comprised adult participants of EMPOWER (n = 942), a phase III clinical trial to evaluate the efficacy of dexpramipexole to treat ALS. Urate was measured in blood samples collected during enrollment as part of the routine block chemistry. We measured outcomes by combined assessment of function and survival rank (CAFs), and time to death, by 12 months. Results showed that in females there was not a significant relation between urate and outcomes...
September 28, 2016: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
https://www.readbyqxmd.com/read/27624123/corticoefferent-pathways-in-pure-lower-motor-neuron-disease-a-diffusion-tensor-imaging-study
#14
Angela Rosenbohm, Hans-Peter Müller, Annemarie Hübers, Albert C Ludolph, Jan Kassubek
Criteria for assessing upper motor neuron pathology in lower motor neuron disease (LMND) still remain major issues in clinical diagnosis. This study was designed to investigate patients with the clinical diagnosis of adult pure LMND by use of whole brain based diffusion tensor imaging (DTI) to delineate alterations of corticoefferent pathways in vivo. Comparison of fractional anisotropy (FA) maps was performed by whole brain-based spatial statistics for 37 LMND patients vs. 53 matched controls to detect white matter structural alterations...
December 2016: Journal of Neurology
https://www.readbyqxmd.com/read/27617773/brain-derived-neurotrophic-factor-facilitates-functional-recovery-from-als-cerebral-spinal-fluid-induced-neurodegenerative-changes-in-the-nsc-34-motor-neuron-cell-line
#15
Shanmukha Shruthi, R Sumitha, Anu Mary Varghese, S Ashok, B K Chandrasekhar Sagar, T N Sathyaprabha, A Nalini, Boris W Kramer, Trichur R Raju, K Vijayalakshmi, Phalguni Anand Alladi
BACKGROUND: The survival of motor neurons is dependent upon neurotrophic factors both during childhood and adolescence and during adult life. In disease conditions, such as in patients with amyotrophic lateral sclerosis (ALS), the mRNA levels of trophic factors like brain-derived neurotrophic factor (BDNF), insulin-like growth factor-1 (IGF-1), fibroblast growth factor-2 (FGF-2), and vascular endothelial growth factor are downregulated. This was replicated in our in vivo experimental system following the injection of cerebral spinal fluid (CSF) of sporadic ALS (ALS-CSF) patients...
September 13, 2016: Neuro-degenerative Diseases
https://www.readbyqxmd.com/read/27589995/phase-i-clinical-trial-of-safety-of-l-serine-for-als-patients
#16
Todd D Levine, Robert G Miller, Walter G Bradley, Dan H Moore, David S Saperstein, Lynne E Flynn, Jonathan S Katz, Dallas A Forshew, James S Metcalf, Sandra A Banack, Paul A Cox
We performed a randomized, double-blind phase I clinical trial for six months on the effects of oral L-serine in patients with ALS. The protocol called for enrollment of patients with a diagnosis of probable or definite ALS, age 18-85 years, disease duration of less than three years and forced vital capacity (FVC) ≥ 60%. Patients were randomly assigned to four different oral twice-daily dose regimens (0.5, 2.5, 7.5, or 15 g/dose). Blood, urine and CSF samples, ALS Functional Rating Scale-Revised (ALSFRS-R) scores and forced vital capacity (FVC) were obtained throughout the trial...
September 2, 2016: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
https://www.readbyqxmd.com/read/27581221/trial-of-early-noninvasive-ventilation-for-als-a-pilot-placebo-controlled-study
#17
Teresa L Jacobs, Devin L Brown, Jonggyu Baek, Erin M Migda, Timothy Funckes, Kirsten L Gruis
OBJECTIVE: To evaluate the use and tolerability of noninvasive positive pressure ventilation (NIV) in patients with amyotrophic lateral sclerosis (ALS) early in their disease by comparing active NIV and sham NIV in patients not yet eligible for NIV use as recommended by practice guidelines. METHODS: This was a single-center, prospective, double-blind, randomized, placebo (sham)-controlled pilot trial. Patients with ALS were randomized to receive either sham NIV or active NIV and underwent active surveillance approximately every 3 months until they reached a forced vital capacity (FVC) <50% or required NIV for clinical symptom management...
November 1, 2016: Neurology
https://www.readbyqxmd.com/read/27567641/the-value-of-magnetic-resonance-imaging-as-a-biomarker-for-amyotrophic-lateral-sclerosis-a-systematic-review
#18
G Grolez, C Moreau, V Danel-Brunaud, C Delmaire, R Lopes, P F Pradat, M M El Mendili, L Defebvre, D Devos
BACKGROUND: Amyotrophic lateral sclerosis (ALS) is a fatal, rapidly progressive neurodegenerative disease that mainly affects the motor system. A number of potentially neuroprotective and neurorestorative disease-modifying drugs are currently in clinical development. At present, the evaluation of a drug's clinical efficacy in ALS is based on the ALS Functional Rating Scale Revised, motor tests and survival. However, these endpoints are general, variable and late-stage measures of the ALS disease process and thus require the long-term assessment of large cohorts...
2016: BMC Neurology
https://www.readbyqxmd.com/read/27552392/what-s-in-the-literature
#19
Nicholas J Silvestri, Gil I Wolfe, David Lacomis, Mark B Bromberg
The Guillain-Barré syndrome (GBS) is one of the few neuropathies well known to the general public, in part because of its association with swine flu vaccinations in 1976. GBS has again reached the general public with its possible association with Zika virus. The virus, borne by infected Aedes aegypti mosquitos, is being linked to birth defects when pregnant women are bitten and infected. There are early reports also linking GBS to Zika infection, which could expose a wider range of infected people to the neuropathy...
September 2016: Journal of Clinical Neuromuscular Disease
https://www.readbyqxmd.com/read/27497700/a-robust-good-manufacturing-practice-compliant-clinical-scale-procedure-to-generate-regulatory-t-cells-from-patients-with-amyotrophic-lateral-sclerosis-for-adoptive-cell-therapy
#20
Abdullah Alsuliman, Stanley H Appel, David R Beers, Rafet Basar, Hila Shaim, Indresh Kaur, Jane Zulovich, Eric Yvon, Muharrem Muftuoglu, Nobuhiko Imahashi, Kayo Kondo, Enli Liu, Elizabeth J Shpall, Katayoun Rezvani
Regulatory T cells (Tregs) play a fundamental role in the maintenance of self-tolerance and immune homeostasis. Defects in Treg function and/or frequencies have been reported in multiple disease models. Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder affecting upper and lower motor neurons. Compelling evidence supports a neuroprotective role for Tregs in this disease. Indeed, rapid progression in ALS patients is associated with decreased FoxP3 expression and Treg frequencies...
October 2016: Cytotherapy
keyword
keyword
93629
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"