Neurology
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Randomized Controlled Trial Multicenter Study
Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS.
To examine the safety and tolerability as well as the preliminary efficacy of arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in patients with rapidly progressive SOD1 amyotrophic lateral sclerosis (ALS). ⋯ This study provides Class II evidence that arimoclomol is safe and well-tolerated at a dosage of 200 mg tid for up to 12 months. The study lacked the precision to conclude, or to exclude, an important therapeutic benefit of arimoclomol.