TY - JOUR
T1 - Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)
AU - Shemesh, Elad
AU - Deroma, Laura
AU - Hendriksz, Christian J.
AU - Hollak, Carla
AU - Krishan, Ashma
N1 - Funding Information:
This project was supported by the National Institute for Health Research, via Cochrane Infrastructure funding to the Cochrane Cystic Fibrosis and Genetic Disorders Group. The views and opinions expressed therein are those of the authors and do not necessarily reflect those of the Systematic Reviews Programme, NIHR, NHS or the Department of Health.
Funding Information:
• National Institute for Health Research, UK. This systematic review was supported by the National Institute for Health Research, via Cochrane Infrastructure funding to the Cochrane Cystic Fibrosis and Genetic Disorders Group.
Publisher Copyright:
© 2018 The Cochrane Collaboration.
PY - 2018/3/12
Y1 - 2018/3/12
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85043513962&partnerID=8YFLogxK
U2 - 10.1002/14651858.CD012961
DO - 10.1002/14651858.CD012961
M3 - Article
AN - SCOPUS:85043513962
SN - 1465-1858
VL - 2018
JO - Cochrane Database of Systematic Reviews
JF - Cochrane Database of Systematic Reviews
IS - 3
M1 - CD012961
ER -