Abstract
Abbreviations and Acronyms:
ALS (amyotrophic lateral sclerosis), ALSFRS-R (ALS Functional Rating Scale-Revised), C9orf72 (chromosome 9 open reading frame 72), FDA (Food and Drug Administration), FTD (frontotemporal dementia), FUS (fused in sarcoma), mRNA (messenger RNA), MSC (mesenchymal stromal cell), SOD1 (copper-zinc superoxide dismutase), TDP-43 (transactive response DNA-binding protein 43)- •No diagnostic test for amyotrophic lateral sclerosis (ALS) is yet available.
- •The prevalence of ALS in the United States is 5 in 100,000.
- •Care for ALS is best delivered through a multidisciplinary ALS clinic.
- •A new intravenous medication, edaravone, was approved for the treatment of ALS in 2017.
- •Edaravone slows disease progression by 33% (measured by the ALS Functional Rating Scale-Revised) in a very select population of patients with ALS.
Background

Epidemiology
Pathogenesis
Clinical Care
Region/involvement | Upper motor neuron suspect findings | Lower motor neuron suspect findings |
---|---|---|
Bulbar | Brainstem lesion (stroke, multiple sclerosis, tumor) | Brainstem lesion (stroke, multiple sclerosis, tumor), neuromuscular junction disorder (myasthenia gravis, muscle-specific tyrosine kinase myasthenia), bulbospinal muscular atrophy |
Cervical | Cervical myelopathy | Multifocal motor neuropathy, cervical radiculopathy |
Lumbosacral | Thoracic myelopathy | Lumbosacral radiculopathy |
- Miller R.G.
- Jackson C.E.
- Kasarskis E.J.
- et al.
- Miller R.G.
- Jackson C.E.
- Kasarskis E.J.
- et al.

Oskarsson B, Moore D, Mozaffar T, et al. Mexiletine for muscle cramps in amyotrophic lateral sclerosis: a randomized double-blind crossover trial [published online March 6, 2018]. Muscle Nerve. https://doi.org/10.1002/mus.26117.
Newly Approved Therapy
Approval letter. Food and Drug Administration; 2017. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/209176Orig1s000Approv.pdf. Accessed May 14, 2018.
Future Therapies
Stem Cells
Other Agents
Conclusion
Supplemental Online Material
References
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Article Info
Publication History
Footnotes
For editorial comment, see page 1543
Potential Competing Interests: B Oskarsson serves as a consultant for FlexPharma and has provided consultations to Biogen regarding SMA. The rest of the authors report no competing interests.