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als
ALS
ALS (30k US; 200k global) is a fatal motor neuron disease with progressive degeneration of upper and lower motor neurons; TDP-43 inclusions are the pathological hallmark in >97%; riluzole and edaravone extend survival modestly; tofersen (SOD1 ASO) is approved for familial ALS.
Entry Metadata
| Field | Value |
|---|---|
| ID | als |
| Name | ALS |
| Status | draft |
| Last reviewed | 2026-06-08 |
| Atlas | 01-human |
| Scale | 07-system |
Cross-Atlas Connections
Sources
- Brown RH, Al-Chalabi A. Amyotrophic lateral sclerosis. N Engl J Med. 2017;377(2):162-172. · PubMed 28700839
- Writing Group on behalf of the Edaravone ALS 19 Study Group. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017;16(7):505-512. · PubMed 28522180
- Miller TM, Cudkowicz ME, Genge A, et al. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099-1110. · PubMed 36129998