Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)

Shemesh, Elad, Deroma, Laura, Hendriksz, Christian J, Hollak, Carla and Krishan, Ashma (2018) Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome). Cochrane Database of Systematic Reviews, 2018 (3).

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Official URL: http://dx.doi.org/10.1002/14651858.cd012961

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.

Item Type:	Article
Depositing User:	Symplectic Admin
Date Deposited:	24 May 2019 15:17
Last Modified:	19 Jan 2023 00:43
DOI:	10.1002/14651858.cd012961
Related URLs:	Publisher
URI:	https://livrepository.liverpool.ac.uk/id/eprint/3042814