title: Quantifying disease progression in amyotrophic lateral sclerosis.
abstract: 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.
authors.affiliation: Department of Neurology, University of California, San Francisco, San Francisco, CA; Prince of Wales Clinical School, University of New South Wales, Randwick, Australia; Neuroscience Research Australia, Barker St, Randwick, Australia.
authors.forename: Neil G
authors.initials: NG
authors.lastname: Simon
authors.forename: Martin R
authors.initials: MR
authors.lastname: Turner
authors.forename: Steve
authors.initials: S
authors.lastname: Vucic
authors.forename: Ammar
authors.initials: A
authors.lastname: Al-Chalabi
authors.forename: Jeremy
authors.initials: J
authors.lastname: Shefner
authors.forename: Catherine
authors.initials: C
authors.lastname: Lomen-Hoerth
authors.forename: Matthew C
authors.initials: MC
authors.lastname: Kiernan
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000235:genetics / Q000473:pathology* / Q000523:psychology / Q000628:therapy; D001921:Brain / Q000473:pathology; D018450:Disease Progression; D006801:Humans; D018482:Muscle, Skeletal / Q000473:pathology / Q000503:physiopathology; D013116:Spinal Cord / Q000473:pathology / Q000503:physiopathology