Improving clinical trial outcomes in amyotrophic lateral sclerosis
Individuals who are diagnosed with amyotrophic lateral sclerosis (ALS) today face the same
historically intransigent problem that has existed since the initial description of the disease in …
historically intransigent problem that has existed since the initial description of the disease in …
Crowdsourcing biomedical research: leveraging communities as innovation engines
The generation of large-scale biomedical data is creating unprecedented opportunities for
basic and translational science. Typically, the data producers perform initial analyses, but it …
basic and translational science. Typically, the data producers perform initial analyses, but it …
Global, regional, and national burden of motor neuron diseases 1990–2016: a systematic analysis for the Global Burden of Disease Study 2016
Background Understanding how prevalence, incidence, and mortality of motor neuron
diseases change over time and by location is crucial for understanding the causes of these …
diseases change over time and by location is crucial for understanding the causes of these …
Cx43 hemichannels contribute to astrocyte-mediated toxicity in sporadic and familial ALS
Connexin 43 (Cx43) gap junctions and hemichannels mediate astrocyte intercellular
communication in the central nervous system under normal conditions and contribute to …
communication in the central nervous system under normal conditions and contribute to …
How data scientistswork together with domain experts in scientific collaborations: To find the right answer or to ask the right question?
In recent years there has been an increasing trend in which data scientists and domain
experts work together to tackle complex scientific questions. However, such collaborations …
experts work together to tackle complex scientific questions. However, such collaborations …
Model-based recursive partitioning for subgroup analyses
The identification of patient subgroups with differential treatment effects is the first step
towards individualised treatments. A current draft guideline by the EMA discusses potentials …
towards individualised treatments. A current draft guideline by the EMA discusses potentials …
Multicentre appraisal of amyotrophic lateral sclerosis biofluid biomarkers shows primacy of blood neurofilament light chain
AG Thompson, E Gray, N Verber, Y Bobeva… - Brain …, 2022 - academic.oup.com
The routine clinical integration of individualized objective markers of disease activity in those
diagnosed with the neurodegenerative disorder amyotrophic lateral sclerosis is a key …
diagnosed with the neurodegenerative disorder amyotrophic lateral sclerosis is a key …
Wearable device and smartphone data quantify ALS progression and may provide novel outcome measures
Amyotrophic lateral sclerosis (ALS) therapeutic development has largely relied on staff-
administered functional rating scales to determine treatment efficacy. We sought to …
administered functional rating scales to determine treatment efficacy. We sought to …
Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS
M Benatar, J Wuu, PM Andersen, N Atassi, W David… - Neurology, 2018 - AAN Enterprises
Objective To examine the safety and tolerability as well as the preliminary efficacy of
arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in …
arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in …
Machine learning in amyotrophic lateral sclerosis: achievements, pitfalls, and future directions
V Grollemund, PF Pradat, G Querin, F Delbot… - Frontiers in …, 2019 - frontiersin.org
Background: Amyotrophic Lateral Sclerosis (ALS) is a relentlessly progressive
neurodegenerative condition with limited therapeutic options at present. Survival from …
neurodegenerative condition with limited therapeutic options at present. Survival from …