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January 21, 2025

Reimagining Care and Research for Amyotrophic Lateral Sclerosis

Author Affiliations
  • 1Department of Neurology, Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston
  • 2Department of Health Policy and Management, Bloomberg School of Public Health, Johns Hopkins University, Baltimore, Maryland
  • 3Department of Neurology, University of Michigan, Ann Arbor
JAMA Neurol. Published online January 21, 2025. doi:10.1001/jamaneurol.2024.4757

Amyotrophic lateral sclerosis (ALS) is a fatal neurological disease involving progressive motor neuron degeneration. While the disease presents as a range of clinical phenotypes, such as bulbar- or spinal-onset ALS, the initial weakness caused by degenerating motor neurons invariably advances to loss of voluntary muscle function and death typically within 2 to 5 years of onset.1 Currently approved treatments do little to slow the disease course, and there is no cure; development of more effective therapies is urgently needed. Drug development in ALS has gained momentum over the past decade since the Ice Bucket Challenge, which raised both awareness and major philanthropic funds for ALS research. Yet this development is hindered by structural, operational, and community engagement gaps that continue to slow the pace of therapeutic discovery for ALS.

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