Motoneuron disease: Clinical

Hristelina Ilieva, Nicholas J Maragakis

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

ALS is a neurodegenerative disease in which the primary symptoms result in progressive neuromuscular weakness. Recent studies have highlighted that there is significant heterogeneity with regard to anatomical and temporal disease progression. Importantly, more recent advances in genetics have revealed new causative genes to the disease. New efforts have focused on the development of biomarkers that could aid in diagnosis, prognosis, and serve as pharmacodynamics markers. Although traditional pharmaceuticals continue to undergo trials for ALS, new therapeutic strategies including stem cell transplantation studies, gene therapies, and antisense therapies targeting some of the familial forms of ALS are gaining momentum.

Original languageEnglish (US)
Title of host publicationAdvances in Neurobiology
PublisherSpringer New York LLC
Pages191-210
Number of pages20
Volume15
DOIs
StatePublished - 2017

Publication series

NameAdvances in Neurobiology
Volume15
ISSN (Print)2190-5215

Keywords

  • Diagnosis
  • Exercise
  • Frontotemporal dementia
  • Gene therapy
  • Heterogeneity
  • Nutrition
  • Phenotype
  • Respiratory management
  • Stem cell therapy

ASJC Scopus subject areas

  • Biochemistry
  • Neurology
  • Developmental Neuroscience
  • Cellular and Molecular Neuroscience

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