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Amyotrophic lateral sclerosis (ALS) exhibits characteristic variability of onset and rate of disease progression, with inherent clinical heterogeneity making disease quantitation difficult. Recent advances in understanding pathogenic mechanisms linked to the development of ALS impose an increasing need to develop strategies to predict and more objectively measure disease progression. This review explores phenotypic and genetic determinants of disease progression in ALS, and examines established and evolving biomarkers that may contribute to robust measurement in longitudinal clinical studies. With targeted neuroprotective strategies on the horizon, developing efficiencies in clinical trial design may facilitate timely entry of novel treatments into the clinic.

Original publication

DOI

10.1002/ana.24273

Type

Journal article

Journal

Ann Neurol

Publication Date

11/2014

Volume

76

Pages

643 - 657

Keywords

Amyotrophic Lateral Sclerosis, Brain, Disease Progression, Humans, Muscle, Skeletal, Spinal Cord