Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)

  1. To assess the effectiveness, safety and appropriate dose regimen of ERT in people with MPS IV A.
  2. 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.
  3. To consolidate recommendations for the design of future clinical trials.

This is a protocol.