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 - 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 -