Advances in the Development of Disease-Modifying Treatments for Amyotrophic Lateral Sclerosis
Diane Moujalled, Anthony R White
CNS DRUGS | ADIS INT LTD | Published : 2016
Amyotrophic 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 glutamate antagonist, riluzole. Seminal breakthroughs in the pathomechanisms and genetic factors associated with ALS have heavily relied on the use of rodent models that recapitulate the ALS phenotype; however, w..View full abstract
Related Projects (7)
DM is supported by an Alzheimer's Australia Dementia Research fellowship (AADRF) and funding from the Motor Neuron Disease Research Institute of Australia (MNDRIA). ARW is supported by an Australian Research Council Future Fellowship and The National Health and Medical Research Council of Australia.