小结
定义
病史和体格检查
诊断性检查
治疗流程
撰稿人
作者
Consultant Haematologist
Royal Free Hospital
Professor in Haematology
University College London
London
UK
利益声明
ABM has received research funding, travel grants for educational meetings, and consultancy fees from Sanofi Genzyme, Shire HGT, Actelion, Biomarin, Protalix, and Amicus. He is also an author of a number of references cited in this topic.
同行评议者
Associate Professor
Departments of Neurology and Pediatrics
NYU School of Medicine
New York
NY
利益声明
GMP declares that he has no competing interests.
Consultant Paediatrician
Paediatric Metabolic Unit
Cambridge University Hospitals
Cambridge
UK
利益声明
UR has received travel grants, honoraria for lectures, and funding for clinical trials from Shire HGT, Genzyme, and Actelion.
Head of Department
Children's Hospital
University of Mainz
Mainz
Germany
利益声明
MB has been reimbursed by Shire, the manufacturer of Elaprase and Replagal, for attending several conferences, for running educational programs and for consulting. MB has received honoraria for speaking from Genzyme (the manufacturer of Myozyme, Fabrazyme, Aldurazyme, and Cerezyme) and Actelion (the manufacturer of Zavesca). MB is an author of a number of references cited in this topic.
Associate Professor of Public Health
Director of Research
Gippsland Medical School
Monash University
Churchill
Australia
利益声明
EVV declares that he has no competing interests.
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