Mucopolysaccharidosis type VI enzyme replacement therapy initiated in adulthood: Findings from the MPS VI clinical surveillance program

Harmatz Paul; Lampe Christina; Parini Rossella; Sharma Reena; Teles Elisa Lea; Hawley Sara; Johnson Julie; Sivam Debbie; Sisic Zlatko

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Mucopolysaccharidosis type VI enzyme replacement therapy initiated in adulthood: Findings from the MPS VI clinical surveillance program

机译：在成年期启动的粘性多族种型VI酶替代疗法：来自MPS VI临床监测计划的调查结果

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来源
《Molecular genetics and metabolism》 |2018年第2期|共1页
作者
Harmatz Paul; Lampe Christina; Parini Rossella; Sharma Reena; Teles Elisa Lea; Hawley Sara; Johnson Julie; Sivam Debbie; Sisic Zlatko;
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作者单位

UCSF Benioff Childrens Hosp Oakland Oakland CA USA;

Helios Dr Horst Schmidt Kliniken Wiesbaden Germany;

AST Fdn MBBM Monza Italy;

Salford Royal Hosp NHS Fdn Trust Salford Lancs England;

Sao Joao Hosp Oporto Portugal;

BioMarin Pharmaceut Inc Novato CA USA;

BioMarin Pharmaceut Inc Novato CA USA;

BioMarin Pharmaceut Inc Novato CA USA;

BioMarin Pharmaceut Inc Novato CA USA;

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原文格式 PDF
正文语种 eng
中图分类内分泌腺疾病及代谢病;
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1. Mucopolysaccharidosis type VI enzyme replacement therapy initiated in adulthood: Findings from the MPS VI clinical surveillance program [J] . Harmatz Paul, Lampe Christina, Parini Rossella, Molecular genetics and metabolism . 2018,第2期

机译：在成年期启动的粘性多族种型VI酶替代疗法：来自MPS VI临床监测计划的调查结果
2. MUCOPOLYSACCHARIDOSIS VI ENZYME REPLACEMENT THERAPY INITIATED IN ADULTHOOD: FINDINGS FROM THE MPS VI CLINICAL SURVEILLANCE PROGRAM [J] . Lampe C., Harmatz P., Parini R., Molecular genetics and metabolism . 2018,第3期

机译：粘性多族菌病VI酶替代治疗在成年期启动：来自国会委员会的临床监测计划的调查结果
3. Enzyme replacement therapy in patients with mucopolysaccharidosis type VI: Updated findings from the MPS VI clinical surveillance program [J] . Harmatz Paul, Lampe Christina, Parini Rossella, Molecular genetics and metabolism . 2019,第2期

机译：粘性多型型患者酶替代疗法VI：MPS VI临床监测计划的更新结果
4. Understanding the Outcome Findings of Cognitive-Behavioral Therapy for Psychosis: A Cognitive Model and Its Clinical Implications [C] . David Fowler, Philippa Garety, Elizabeth Kuipers Keio University International Symposium for Life Sciences and Medicine . 2002

机译：了解精神病认知行为治疗的结果结果：认知模型及其临床意义
5. Human hematopoietic progenitor cell-directed gene therapy in a murine xenotransplantation model of mucopolysaccharidosis type VII. [D] . Hofling, August Alexander. 2005

机译：人类造血祖细胞指导的基因治疗在粘多糖贮积症类型VII的小鼠异种移植模型中。
6. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy [O] . J. Edmond Wraith, Maurizio Scarpa, Michael Beck, -1

机译：II型粘多糖贮积病（Hunter综合征）：酶替代疗法时代的临床回顾和治疗建议
7. Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program [O] . Paul R. Harmatz, Christina Lampe, Rossella Parini, 2019

机译：酶替代疗法患有疾病谱的结果：来自粘性族多种子胞菌的结果VI临床监督计划

Mucopolysaccharidosis type VI enzyme replacement therapy initiated in adulthood: Findings from the MPS VI clinical surveillance program

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