Biomarkers in amyotrophic lateral sclerosis

Turner, Martin R; Kiernan, Matthew C.; Leigh, P. Nigel; Talbot, Kevin

doi:10.1016/s1474-4422(08)70293-x

Cited by 399 publications

(315 citation statements)

References 123 publications

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“…We should mention some limitations of our study: first, the relatively small sample size, which was nevertheless comparable with previous reports. 7 Due to the exploratory nature of our study, we did not correct for multiple comparisons; therefore, some of the significances might have been overestimated. However, we observed DTI tractography changes only in brain areas that are known to be the preferential sites of ALS damage from neuropathologic studies.…”

Section: Discussionmentioning

confidence: 99%

“…58 Despite such limitations, this study highlights the potential of DTI of the brain to provide in vivo markers of cerebral involvement in ALS. 7 …”

Section: Discussionmentioning

confidence: 99%

“…5,6 Most DTI studies in ALS have been limited to the assessment of the CST damage. 7 Whether on the basis of regions of interest, [8][9][10][11][12][13][14][15][16] voxel-based analysis, [17][18][19][20] or tractography, [21][22][23] DTI studies in ALS have shown reduced FA along the CST.…”

mentioning

confidence: 99%

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Assessment of White Matter Tract Damage in Patients with Amyotrophic Lateral Sclerosis: A Diffusion Tensor MR Imaging Tractography Study: Fig 1.

Agosta¹,

Pagani²,

Petrolini³

et al. 2010

AJNR Am J Neuroradiol

138

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Section: Discussionmentioning

confidence: 99%

“…58 Despite such limitations, this study highlights the potential of DTI of the brain to provide in vivo markers of cerebral involvement in ALS. 7 …”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Assessment of White Matter Tract Damage in Patients with Amyotrophic Lateral Sclerosis: A Diffusion Tensor MR Imaging Tractography Study: Fig 1.

Agosta¹,

Pagani²,

Petrolini³

et al. 2010

AJNR Am J Neuroradiol

138

View full text Add to dashboard Cite

show abstract

“…Biofluid-derived [16][17][18], imaging-based, and electrophysiologic ALS biomarkers are all currently in development or validation. Phosphorylated neurofilament heavy chain appears elevated at stable levels throughout the disease [19][20][21][22][23][24][25][26].…”

Section: Toward Novel Outcome Measures and Biomarkers In Als Trialsmentioning

confidence: 99%

Clinical Trial Designs in Amyotrophic Lateral Sclerosis: Does One Design Fit All?

2015

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The last 2 decades have seen a surge in the number of amyotrophic lateral sclerosis (ALS) clinical trials with the hope of finding successful treatments. Clinical trialists aim to repurpose existing drugs and test novel compounds to target potential ALS disease pathophysiology. Recent technological advancements have led to the discovery of new causative genetic agents and modes of delivering potential therapy, calling for increasingly sophisticated trial design. The standard ALS clinical trial design may be modified depending on study needs: type of therapy; route of therapy delivery; phase of therapy development; applicable subpopulation; market availability of therapy; and utility of telemedicine. Novel biomarkers of diagnostic, predictive, prognostic, and pharmacodynamic value are undergoing development and validation for use in clinical trials. Design modifications build on the traditional clinical trial design and may be employed in either the learning or confirming trial phase. Novel designs aim to minimize patient risk, study duration, and sample size, while improving efficiency and promoting statistical power to herald an exciting era for clinical research in ALS.

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“…The motor function phenotypes of ALS are highly heterogeneous and determined by three primary independent attributes: body region of onset; relative mix of UMN and LMN involvement; rate of progression [2]. Phenotypes that predominantly affect specific body regions are associated with extremes of disease progression; for example, patients with bulbar onset are typically associated with a more rapid decline [2,3]. The cause of ALS is generally unknown, although approximately 2% of cases are due to mutations in the superoxide dismutase gene [4].…”

Section: Introductionmentioning

confidence: 99%

Neuroimaging in Amyotrophic Lateral Sclerosis

et al. 2011

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Summary: Amyotrophic lateral sclerosis (ALS) is a motor neuron disease characterized by progressive degeneration of upper motor neurons (UMN) and lower motor neurons (LMN). While LMN dysfunction can be confirmed by electromyography (EMG) and muscle biopsy, UMN involvement is more difficult to detect, particularly in the early phase. Objective and sensitive measures of UMN dysfunction are needed for early diagnosis and monitoring of disease progression and therapeutic efficacy. Advanced magnetic resonance imaging (MRI) techniques, such as diffusion, perfusion, magnetization transfer imaging, functional MRI, and MR spectroscopy, provide insight into the pathophysiological processes of ALS and may have a role in the identification and monitoring of UMN pathology. This article provides an overview of these neuroimaging techniques and their potential roles in ALS.

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Biomarkers in amyotrophic lateral sclerosis

Cited by 399 publications

References 123 publications

Assessment of White Matter Tract Damage in Patients with Amyotrophic Lateral Sclerosis: A Diffusion Tensor MR Imaging Tractography Study: Fig 1.

Assessment of White Matter Tract Damage in Patients with Amyotrophic Lateral Sclerosis: A Diffusion Tensor MR Imaging Tractography Study: Fig 1.

Clinical Trial Designs in Amyotrophic Lateral Sclerosis: Does One Design Fit All?

Neuroimaging in Amyotrophic Lateral Sclerosis

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