Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)

Elad Shemesh, Laura Deroma, Christian J. Hendriksz, Carla Hollak, Ashma Krishan

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


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.

Original languageEnglish
Article numberCD012961
JournalCochrane Database of Systematic Reviews
Issue number3
StatePublished - 12 Mar 2018
Externally publishedYes

ASJC Scopus subject areas

  • Pharmacology (medical)


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