Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)

Abstract

This is a protocol for a Cochrane Review (Intervention). The objectives are as follows: To assess the effectiveness, safety and appropriate dose regimen of ERT in people with MPS IV A. To determine whether evidence from NRSIs (which potentially offers longer follow-ups) can contribute to the ERT efficacy evidence-base, and to determine the potential need for additional RCT evidence. To consolidate recommendations for the design of future clinical trials.