Hereditary Motor Neuropathies and Amyotrophic Lateral Sclerosis: a Molecular and Clinical Update

Rocio Garcia-Santibanez, Matthew Burford, Robert C. Bucelli

Research output: Contribution to journalReview article

5 Scopus citations

Abstract

Purpose of Review: This article provides an overview of recent advancements in the fields of hereditary motor neuropathies and ALS. Recent Findings: There has been a robust growth in our knowledge and understanding of hereditary and degenerative motor neuronopathies/neuropathies over the last decade. Many breakthroughs in the field of hereditary motor neuropathies (HMN) have been associated with identification and characterization of the genes and molecular mechanisms underlying these disorders. Similar recent breakthroughs on the genetic and molecular underpinnings of the degenerative motor neuronopathy, amyotrophic lateral sclerosis (ALS), have been accompanied by advancements in biomarker research and the development and FDA approval of novel therapies. Summary: There is a reasonable hope that the marked and continued growth in our understanding of the molecular pathophysiology of the HMNs will translate into novel therapeutic approaches in the decade to come. Such breakthroughs have already begun in ALS, where novel biomarkers and treatment strategies have translated into a new FDA-approved therapy with a number of promising agents in development and/or in definitive phase 2/3 trials.

Original languageEnglish
Article number93
JournalCurrent neurology and neuroscience reports
Volume18
Issue number12
DOIs
StatePublished - Dec 1 2018

Keywords

  • Amyotrophic lateral sclerosis
  • Antisense oligonucleotides
  • C9orf72
  • Edaravone
  • Hereditary motor neuropathy
  • SOD1

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