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Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)

This is a protocol for a Cochrane Review (Intervention). The objectives are as follows: 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 E...

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Detaylı Bibliyografya
Yayımlandı:Cochrane Database Syst Rev
Asıl Yazarlar: Shemesh, Elad, Deroma, Laura, Hendriksz, Christian J, Hollak, Carla, Krishan, Ashma
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: John Wiley & Sons, Ltd 2018
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6494408/
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/14651858.CD012961
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