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Disease modifying treatment of amyotrophic lateral sclerosis

Rabia B Choudry, MD
Nestor Galvez-Jimenez, MD, MSc, MHSA, FACP
Merit E Cudkowicz, MD, MSc
Section Editors
Jeremy M Shefner, MD, PhD
Ira N Targoff, MD
Deputy Editor
John F Dashe, MD, PhD


Amyotrophic lateral sclerosis (ALS), first described by Charcot in the nineteenth century [1], is a progressive neurodegenerative disorder that causes muscle weakness, disability, and eventually death, with a median survival of three to five years.

The hallmark of ALS is the combination of upper motor neuron (UMN) and lower motor neuron (LMN) involvement. The LMN findings of weakness, atrophy, and fasciculations are a direct consequence of muscle denervation. The UMN findings of hyperreflexia and spasticity result from degeneration of the lateral corticospinal tracts in the spinal cord [1].

The existing and experimental disease modifying pharmacologic treatment of ALS will be reviewed here. The symptomatic management and the clinical features and diagnosis of ALS are discussed separately. (See "Symptom-based management of amyotrophic lateral sclerosis" and "Clinical features of amyotrophic lateral sclerosis and other forms of motor neuron disease" and "Diagnosis of amyotrophic lateral sclerosis and other forms of motor neuron disease".)


Riluzole is the only drug to have any impact on survival in ALS [2]. The evidence that riluzole is beneficial comes from two landmark clinical trials [3,4]:

In a prospective, double-blind, placebo-controlled trial in 155 outpatients with ALS, survival at 12 months was significantly higher for patients receiving riluzole (100 mg/day) compared with controls (74 versus 58 percent) [3]. For the subset of patients with bulbar-onset ALS, an even greater advantage for survival at 12 months emerged for the riluzole group (73 versus 35 percent).


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Literature review current through: Sep 2016. | This topic last updated: Sep 23, 2016.
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