PO247 Dissemination bias in clinical trials of neurodegenerative diseases: an observational study

2017 ◽  
Vol 88 (Suppl 1) ◽  
pp. A77.3-A77
Author(s):  
James D Stefaniak ◽  
Thomas CH Lam ◽  
Naomi E Sim ◽  
Rustam Al-Shahi Salman ◽  
David P Breen
2021 ◽  
Vol 14 (3) ◽  
pp. 281
Author(s):  
Francesco Oddone ◽  
Luca Rossetti ◽  
Mariacristina Parravano ◽  
Diego Sbardella ◽  
Massimo Coletta ◽  
...  

Cytidine 5’-diphosphocholine has been widely studied in systemic neurodegenerative diseases, like Alzheimer’s disease, Parkinson’s disease, and brain ischemia. The rationale for the use of citicoline in ophthalmological neurodegenerative diseases, including glaucoma, anterior ischemic optic neuropathy, and diabetic retinopathy, is founded on its multifactorial mechanism of action and the involvement in several metabolic pathways, including phospholipid homeostasis, mitochondrial dynamics, as well as cholinergic and dopaminergic transmission, all being involved in the complexity of the visual transmission. This narrative review is aimed at reporting both pre-clinical data regarding the involvement of citicoline in such metabolic pathways (including new insights about its role in the intracellular proteostasis through an interaction with the proteasome) and its effects on clinical psychophysical, electrophysiological, and morphological outcomes following its use in ophthalmological neurodegenerative diseases (including the results of the most recent prospective randomized clinical trials).


2019 ◽  
Vol 71 (2) ◽  
pp. 415-418 ◽  
Author(s):  
Kwonjune J Seung ◽  
Palwasha Khan ◽  
Molly F Franke ◽  
Saman Ahmed ◽  
Stalbek Aiylchiev ◽  
...  

Abstract Delamanid should be effective against highly resistant strains of Mycobacteriumtuberculosis, but uptake has been slow globally. In the endTB (expand new drug markets for TB) Observational Study, which enrolled a large, heterogeneous cohorts of patients receiving delamanid as part of a multidrug regimen, 80% of participants experienced sputum culture conversion within 6 months. Clinical Trials Registration. NCT02754765.


2018 ◽  
Vol 104 ◽  
pp. 35-45 ◽  
Author(s):  
Diana Jurić ◽  
Shelly Pranić ◽  
Ružica Tokalić ◽  
Ana Marija Milat ◽  
Ivana Mudnić ◽  
...  

Author(s):  
Rosanna Tortelli ◽  
Davide Seripa ◽  
Francesco Panza ◽  
Vincenzo Solfrizzi ◽  
Giancarlo Logroscino

Author(s):  
Jeffrey L. Cummings ◽  
Jagan A. Pillai

Neurodegenerative diseases (NDDs) are growing in frequency and represent a major threat to public health. Advances in scientific progress have made it clear that NDDs share many underlying processes, including shared intracellular mechanisms such as protein misfolding and aggregation, cell-to-cell prion-like spread, growth factor signaling abnormalities, RNA and DNA disturbances, glial cell changes, and neuronal loss. Transmitter deficits are shared across many types of disorders. Means of studying NDDs with human iPS cells and transgenic models are similar. The progression of NDDs through asymptomatic, prodromal, and manifest stages is shared across disorders. Clinical features of NDDs, including cognitive impairment, disease progression, age-related effects, terminal stages, neuropsychiatric manifestations, and functional disorders and disability, have many common elements. Clinical trials, biomarkers, brain imaging, and regulatory aspects of NDD can share information across NDDs. Disease-modifying and transmitter-based therapeutic interventions, clinical trials, and regulatory approaches to treatments for NDDs are also similar.


Author(s):  
Jeremy Prout ◽  
Tanya Jones ◽  
Daniel Martin

This chapter summarizes some aspects of study design and statistical analysis to allow the anaesthetist to appraise research. Types of observational study are described and aspects of interventional studies such as sample size calculation and power are explained. Research governance, phases of drug trials and levels of evidence are described. A section on statistical analysis includes expression of proportion for binary data (odds ratio, number needed to treat) and use of probability and confidence intervals to measure statistical significance.


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