Disease modifying treatment of amyotrophic lateral sclerosis
- Rabia B Choudry, MD
Rabia B Choudry, MD
- Attending Neurologist
- Albert Einstein Medical Center
- Nestor Galvez-Jimenez, MD, MSc, MHSA, FACP
Nestor Galvez-Jimenez, MD, MSc, MHSA, FACP
- The Pauline M. Braathen Endowed Chair
- Chairman, Department of Neurology
- Director, Pauline M. Braathen Neuroscience Center at Neurological Institute Cleveland Clinic.
- Chief, Movement Disorders Program
- Cleveland Clinic Florida
- Clinical Professor of Neurology
- Herbert Wertheim College of M
- Merit E Cudkowicz, MD, MSc
Merit E Cudkowicz, MD, MSc
- Professor of Neurology
- Harvard Medical School
- Section Editors
- Jeremy M Shefner, MD, PhD
Jeremy M Shefner, MD, PhD
- Section Editor — Neuromuscular Disease
- Professor and Chair of Neurology, Barrow Neurological Institute
- Professor of Neurology, University of Arizona, Phoenix
- Clinical Professor of Neurology, Creighton University
- Ira N Targoff, MD
Ira N Targoff, MD
- Section Editor — Muscle Disease
- Professor of Medicine, Section of Rheumatology
- University of Oklahoma Health Sciences Center
Amyotrophic lateral sclerosis (ALS), first described by Charcot in the nineteenth century , 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 .
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".)
●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) . 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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