Huntington's Disease Clinical Trials Corner: April 2022

被引：19

作者：

Estevez-Fraga, Carlos ^{[1
]}

Rodrigues, Filipe B. ^{[1
,2
,3
]}

Tabrizi, Sarah J. ^{[1
]}

Wild, Edward J. ^{[1
,4
]}

机构：

[1] UCL Queen Sq Inst Neurol, Huntingtons Dis Ctr, London, England

[2] Univ Lisbon, Fac Med, Lab Clin Pharmacol & Therapeut, Lisbon, Portugal

[3] Inst Med Mol, Lisbon, Portugal

[4] UCL Huntingtons Dis Ctr, 2nd Floor,Russell Sq House,10-12 Russell Sq, London WC1B 5EH, England

来源：

JOURNAL OF HUNTINGTONS DISEASE | 2022年 / 11卷 / 02期

基金：

英国惠康基金; 英国医学研究理事会;

关键词：

Huntington disease; clinical trials;

D O I：

10.3233/JHD-229002

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

In this edition of the Huntington's Disease Clinical Trials Corner we expand on GENERATION HD1, PRECISION-HD1 and PRECISION-HD2, SELECT-HD, and VIBRANT-HD trials, and list all currently registered and ongoing clinical trials in Huntington's disease.

引用

页码：105 / 118

页数：14

共 24 条

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Effenberger, Kerstin A. ;

Woll, Matthew G. ;

Jani, Minakshi B. ;

Risher, Nicole ;

Yeh, Shirley ;

Cheng, Yaofeng ;

Sydorenko, Nadiya ;

Moon, Young-Choon ;

Karp, Gary M. ;

Weetall, Marla ;

Dakka, Amal ;

Gabbeta, Vijayalakshmi ;

Naryshkin, Nikolai A. ;

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NATURE COMMUNICATIONS, 2021, 12 (01)

[2]

Biogen, 2018, REACTIONS WEEKLY, V1714, P3, DOI [10.1007/s40278-018-50183-2, DOI 10.1007/S40278-018-50183-2]

[3]

Boak L., 2022, CHDI FDN ANN THERAPE

[4] Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry [J].

Kay, Chris ;

Collins, Jennifer A. ;

Skotte, Niels H. ;

Southwell, Amber L. ;

Warby, Simon C. ;

Caron, Nicholas S. ;

Doty, Crystal N. ;

Nguyen, Betty ;

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Ross, Colin J. ;

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Hayden, Michael R. .

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[5] An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion [J].

Keller, Caroline Gubser ;

Shin, Youngah ;

Monteys, Alex Mas ;

Renaud, Nicole ;

Beibel, Martin ;

Teider, Natalia ;

Peters, Thomas ;

Faller, Thomas ;

St-Cyr, Sophie ;

Knehr, Judith ;

Roma, Guglielmo ;

Reyes, Alejandro ;

Hild, Marc ;

Lukashev, Dmitriy ;

Theil, Diethilde ;

Dales, Natalie ;

Cha, Jang-Ho ;

Borowsky, Beth ;

Dolmetsch, Ricardo ;

Davidson, Beverly L. ;

Sivasankaran, Rajeev .

NATURE COMMUNICATIONS, 2022, 13 (01)

[6]

Novartis Pharmaceuticals, 2021, A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease (VIBRANT-HD)

[7]

Palacino J, 2015, NAT CHEM BIOL, V11, P511, DOI [10.1038/NCHEMBIO.1837, 10.1038/nchembio.1837]

[8]

Roche Hoffman-La., 2019, STUD EV EFF SAF INTR

[9] Huntington's Disease Clinical Trials Corner: April 2020 [J].

Rodrigues, Filipe B. ;

Wild, Edward J. .

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[10] Huntington's Disease Clinical Trials Corner: June 2019 [J].

Rodrigues, Filipe B. ;

Ferreira, Joaquim J. ;

Wild, Edward J. .

JOURNAL OF HUNTINGTONS DISEASE, 2019, 8 (03) :363-371

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