Transport®NPC: open phase 3 global trial of intravenous hydroxypropyl-beta-cyclodextrin in patients with Niemann-Pick disease type C1 (NPC1)

被引：0

作者：

Hastings, Caroline A. ^{[1
]}

Goker-Alpan, Ozlem ^{[2
]}

Pena, Loren ^{[3
]}

Bayarri, Jordi Gascon ^{[4
]}

Martin-Hernandez, Elena ^{[5
]}

Sharma, Reena ^{[6
]}

Chacham, Orna Staretz ^{[7
]}

Spiegel, Ronen ^{[8
]}

Mengel, Eugen ^{[9
]}

Walterfang, Mark ^{[10
]}

Peters, Heidi ^{[11
]}

Fung, Victor S. ^{[12
]}

Kiec-Wilk, Beata ^{[13
]}

Smith, Nicholas ^{[14
]}

Giugliani, Roberto ^{[15
]}

Guelbert, Norberto ^{[16
]}

Ezgu, Fatih ^{[17
]}

Ucar, Sema Kalkan ^{[18
]}

Murray, Bryan J. ^{[19
]}

Brecht, Andreas F. ^{[20
]}

Mejia, Joseph ^{[21
]}

机构：

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

[2] Lysosomal Rare Disorders Res & Treatment Ctr Inc, Fairfax, VA USA

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

[4] Bellvitge Univ Hosp, Barcelona, Spain

[5] Hosp Univ 12 Octubre, Madrid, Spain

[6] Salford Royal Hosp NHS Fdn Trust, Salford, Lancs, England

[7] Ben Gurion Univ Negev, Beer Sheva, Israel

[8] Emek Med Ctr, Afula, Israel

[9] SpinCS GmbH, Hochheim, Germany

[10] Royal Melbourne Hosp, Melbourne, Australia

[11] Royal Childrens Hosp, Melbourne, Australia

[12] Westmead Hosp, Westmead, NSW, Australia

[13] Szpital Uniwersytecki Krakowie, Krakow, Poland

[14] Womens & Childrens Hlth Network, Adelaide, SA, Australia

[15] Univ Fed Rio Grande do Sul, Porto Alegre, RS, Brazil

[16] Hosp Ninos Cordoba, Buenos Aires, Argentina

[17] Gazi Univ, Ankara, Turkiye

[18] Ege Univ, Med Sch, Irmir, Turkiye

[19] Boyd Consultants, St Neots, England

[20] Cyclo Therapeut Ltd, Hemmiken, Switzerland

[21] Cyclo Therapeut, Gainesville, FL USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2023.107875

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

136

引用

页数：2

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