Cochrane Database of Systematic Reviews Protocol - Intervention
Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)
Abstract
This is a protocol for a Cochrane Review (Intervention). The objectives are as follows:
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To assess the effectiveness, safety and appropriate dose regimen of ERT in people with MPS IV A.
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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.
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To consolidate recommendations for the design of future clinical trials.
