Defining pre-symptomatic amyotrophic lateral sclerosis

Michael G Benatar, Martin R. Turner, Joanne Wuu

Research output: Contribution to journalReview article

3 Citations (Scopus)

Abstract

Successful treatment of neurodegenerative disease may hinge on early therapeutic intervention. This requires an understanding of early/pre-symptomatic disease, a need that is underscored by advances in antisense oligonucleotide, and viral-vector-based gene therapies. In amyotrophic lateral sclerosis (ALS), the study of pre-symptomatic disease requires a cohesive conceptual framework for describing this phase of disease. Informed by the literature in other neurodegenerative diseases and extensive personal experience, a model is proposed that distinguishes ALS as a clinical syndrome from ALS as a disease, and characterizes pre-symptomatic ALS as having two identifiable stages: pre-manifest and prodromal. The unique and critical importance of biomarker development is articulated and an operational definition of phenoconversion is provided. It is hoped that this framework will accelerate collective efforts to study pre-symptomatic ALS, and aid in the design and implementation of an early intervention- or disease-prevention trial.

Fingerprint

Amyotrophic Lateral Sclerosis
Asymptomatic Diseases
Neurodegenerative Diseases
Antisense Oligonucleotides
Genetic Therapy
Biomarkers
Therapeutics

Keywords

  • biomarkers
  • pre-manifest
  • Pre-symptomatic
  • prodromal

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

Cite this

Defining pre-symptomatic amyotrophic lateral sclerosis. / Benatar, Michael G; Turner, Martin R.; Wuu, Joanne.

In: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 01.01.2019.

Research output: Contribution to journalReview article

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