Advances in the Development of Disease-Modifying Treatments for Amyotrophic Lateral Sclerosis

  title={Advances in the Development of Disease-Modifying Treatments for Amyotrophic Lateral Sclerosis},
  author={Diane Moujalled and A. White},
  journal={CNS Drugs},
  • Diane Moujalled, A. White
  • Published 2016
  • Medicine
  • CNS Drugs
  • AbstractAmyotrophic lateral sclerosis (ALS) is a progressive adult-onset, neurodegenerative disease characterized by the degeneration of upper and lower motor neurons. Over recent years, numerous genes ha ve been identified that promote disease pathology, including SOD1, TARDBP, and the expanded hexanucleotide repeat (GGGGCC) within C9ORF72. However, despite these major advances in identifying genes contributing to ALS pathogenesis, there remains only one currently approved therapeutic: the… CONTINUE READING
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