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Drug treatment for spinal muscular atrophy types II and III

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Abstract

This is a protocol for a Cochrane Review (Intervention). The objectives are as follows:

To review systematically the evidence from randomised controlled trials concerning the efficacy and safety of any drug therapy designed to slow or arrest the progress of the disease in SMA type II and type III. We will perform a separate Cochrane review on spinal muscular atrophy type I.