Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis is a progressive and fatal neurodegenerative disorder of upper and lower motor neurons. No curative therapies exist. Two oral medications, riluzole and edaravone, are approved by the FDA and modestly decrease disease progression in sporadic ALS. Tofersen, an intrathecally administered gene-based therapy, is also FDA approved and slows disease progression in patients with pathogenic gene variants.

Corresponding Author: John Ravits, MD, Altman Clinical Translational Institute, University of California, San Diego, 9452 Medical Center Dr, Ste 2W-108, La Jolla, CA 92037 (jravits@health.ucsd.edu).

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