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Quantifying disease progression in amyotrophic lateral sclerosis
Author(s) -
Simon Neil G.,
Turner Martin R.,
Vucic Steve,
AlChalabi Ammar,
Shefner Jeremy,
LomenHoerth Catherine,
Kiernan Matthew C.
Publication year - 2014
Publication title -
annals of neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.764
H-Index - 296
eISSN - 1531-8249
pISSN - 0364-5134
DOI - 10.1002/ana.24273
Subject(s) - amyotrophic lateral sclerosis , disease , clinical trial , medicine , neuroprotection , clinical study design , neuroscience , physical medicine and rehabilitation , psychology , pathology
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. Ann Neurol 2014;76:643–657