Impact of two-year elosulfase alfa treatment on patient-reported outcomes in patients with Morquio syndrome type A: Results from an English managed access agreement

被引：1

作者：

Adam, Jacqueline ^{[1
]}

Cleary, Maureen ^{[2
]}

Hendriksz, Christian ^{[3
]}

Geberhiwot, Tarekegn ^{[4
]}

Hughes, Derralynn ^{[5
]}

Jones, Simon A. ^{[6
]}

Jovanovic, Ana ^{[7
]}

Morrison, Alexandra ^{[1
]}

Murphy, Elaine ^{[8
]}

Vijay, Suresh ^{[9
]}

机构：

[1] MPS Commercial, Amersham, England

[2] Great Ormond St Hosp Sick Children, London, England

[3] Univ Pretoria, Sch Med, Pretoria, South Africa

[4] New Queen Elizabeth Hosp, Birmingham, W Midlands, England

[5] Royal Free London NHS FT & UC, London, England

[6] Univ Manchester, Manchester, Lancs, England

[7] Salford Royal NHS Fdn Trust, Salford, Lancs, England

[8] Natl Hosp Neurol & Neurosurg, London, England

[9] Birmingham Childrens Hosp NHS Fdn Trust, Birmingham, W Midlands, England

来源：

关键词：

D O I：

10.1016/j.ymgme.2018.12.022

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：S19 / S19

页数：1