A new randomized placebo-controlled study to establish the safety and efficacy of velmanase alfa (human recombinant alpha-mannosidase) enzyme replacement therapy for the treatment of alpha-mannosidosis

被引：0

作者：

Harmatz, Paul ^{[1
]}

Jayakar, Parul ^{[2
]}

Pena, Loren D. M. ^{[3
,4
]}

Cattaneo, Federica ^{[5
]}

Ceravolo, Ferdinando ^{[5
]}

Paleari, Ylenia ^{[5
]}

Sutton, Vernon R. ^{[6
]}

Burton, Barbara K. ^{[7
,8
]}

Longo, Nicola ^{[9
]}

机构：

[1] UCSF Benioff Childrens Hosp, Oakland, CA USA

[2] Nicklaus Childrens Hosp, Miami, FL USA

[3] Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA

[4] Univ Cincinnati, Cincinnati, OH USA

[5] Chiesi Farmaceut SpA, Parma, Italy

[6] Baylor Coll Med, Houston, TX 77030 USA

[7] Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA

[8] Northwestern Univ, Feinberg Sch Med, Chicago, IL 60611 USA

[9] Univ Utah, Salt Lake City, UT USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2020年 / 129卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2019.11.166

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

157

引用

页码：S70 / S70

页数：1

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[1] Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis
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[10] Global treatment response analysis of velmanase alfa long term enzyme replacement therapy for alpha-mannosidosis shows treatment benefit across ages
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Tylki-Szymanska, Anna
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De Meirleir, Linda
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Dali, Christine I.
Jones, Simon A.
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Guffon, Nathalie
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