Motor neuron disease: systematic reviews of treatment for ALS and SMA

Abstract

IntroductionThere is no curative treatment for the common motor neuron diseases, amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy. Nevertheless, there is an increasing volume of published studies. This review assesses the current evidence for treatment of these conditions.Sources of dataPrimarily, the systematic reviews of the Cochrane Collaboration, with additional reference to other systematic reviews and online sites.Areas of agreementRiluzole remains the only medication with demonstrated efficacy and regulatory approval for the treatment of ALS.Areas of controversy, growing points and areas timely for developing researchThe design of clinical trials and the publication of unsatisfactory studies, in both human and animal models, continue to cause confusion in advising on patient management. Improvements in trial design, critical assessment of studies for publication and avoidance of bias towards publication of positive results are needed. A better understanding of pathogenesis should lead to more potent interventions.